qanastek/EMEA-V3 huggingface dataset:
Monitoring betaine plasma concentrations does not help to define the efficacy of treatment, since these concentrations do not directly correspond to the flux through the cytosolic betaine homocysteine methyl transferase pathway.
Monitoraġġ ta ’ konċentrazzjonijiet ta ’ betaine fil- plażma ma jgħinx biex jiġu mfissra l- effikaċja tat - trattament, billi dawn il- konċentrazzjonijiet ma jikkorrispondux direttament mal- fluss minn ġol - mogħdija cytosolic betaine homocysteine methyl transferase.
The absolute bioavailability of betaine has not been determined.
Il- bijodisponibilità ta 'betaine ma ġietx stabbilita.
In healthy adult volunteers (age between 21 to 49 years), after a single oral dose of betaine (50 mg/ kg), absorption was rapid (tmax = 0.9 ± 0.3 hours and a Cmax = 0.9 ± 0.2 mM).
F’ voluntieri adulti f’ saħħithom (età ta ’ bejn 21 u 49 sena), wara doża waħdanija orali ta ’ betaine (50 mg/ kg), l- assorbiment kien rapidu( tmax = 0. 9 ± 0. 3 sigħat u Cmax = 0. 9 ± 0. 2 mM).
After a repeated dose regimen of 100 mg/ kg/ day for 5 days, the absorption kinetics did not change but the distribution half life was prolonged significantly (up to 36 h), indicating saturable transport and redistribution processes.
Betaine kien distribwit rapidament għal ġo volum relattivament kbir (V/ F = 1. 3 l/ kg), b’ rata t’ eliminazzjoni bil- mod (half life medja = 14- il siegħa b' rata ta 'eliminazzjoni totali mill- ġisem medja, CL/ F, = 84 ml/ h/ kg), eliminazzjoni renali meqjusa negliġibbli (5% ta ’ l - eliminazzjoni totali mill- ġisem), fuq preżunzjoni ta' bijodisponibbiltà ta ’ 100% Wara reġimen ta ’ doża ripetuta ta ’ 100 mg/ kg/ kuljum għal 5 ijiem, il- kinetiċi ta ’ l- assorbiment ma nbidilx imma l- half life tad- distribuzzjoni kienet imtawwla sinifikattivament (sa 36 siegħa), li jindika trasport saturat u proċessi ta ’ distribuzzjoni mill- ġdid.
The pharmacokinetic data of homocystinuric patients on long-term betaine supplementation are very similar to those of healthy volunteers.
Id- dejta farmakokinetika ta ’ pazjenti omoċistinuriċi fuq supplimentazzjoni ta ’ betaine għal tul ta ’ żmien huma simili għal dawk ta ’ voluntieri f’ saħħithom.
This demonstrates that differences in betaine kinetics are most probably due to betaine depletion in untreated homocystinuria and are only meaningful for the initial treatment.
Dan juri li differenzi fil- kinetika ta ’ betaine huma wisq probabbli dovuti għal tnaqqis ta ’ betaine f’ omoċistinurja mhux ittrattata u huma sinifikanti biss għat- trattament tal- bidu.
At high doses, a CNS depressant effect and irritation of the gastrointestinal tract was seen in rats.
F’ dożi għoljin, effett dipressanti tas- SNĊ u irritazzjoni tal- passaġġ gastrointestinali kienu murija fil - firien.
Long-term carcinogenicity and reproductive toxicity studies have not been conducted on betaine.
Karċinoġeniċittà u studji riproduttivi tossiċi ma ġewx magħmula fuq betaine.
A standard battery of genotoxicity test reveals no specific hazard for humans.
Batterija standard ta ’ testijiet ġenotossiċi ma wriet l- ebda periklu għal bnedmin.
None.
Xejn
5 After the first opening:
Wara l- ewwel ftuħ:
1 month.
Xahar.
Do not store above 25°C.
Taħżinx f' temperatura ogħla minn 25°C.
Keep the bottle tightly closed in order to protect from moisture.
Żomm il- flixkun magħluq sew sabiex tipproteġi mill- umdità.
HDPE bottles with a child resistant closure.
Fliexken ta 'l- HDPE b' għeluq li hu reżistenti għat- tfal.
Each pack contains 1 bottle with 180 g of powder.
Kull pakkett fih flixkun b' 180 g ta 'trab.
Three measuring spoons are included in each pack.
Tliet kuċċarini bħala miżuri huma inklużi f' kull pakkett.
Orphan Europe SARL Immeuble “ Le Wilson” F - 92058 Paris-La Défense France
Orphan Europe SARL Immeuble “ Le Wilson ” F – 92058 Paris- La Défense Franza
15/ 02/ 2007
15/ 02/ 20007
DATE OF REVISION OF THE TEXT
DATA TA 'META ĠIET RIVEDUTA DIN IL- KITBA
MANUFACTURING AUTHORISATION HOLDER RESPONSIBLE FOR BATCH RELEASE
DETENTUR( I) TA ’ L- AWTORIZZAZZJONI TAL - MANIFATTURA RESPONSABBLI GĦALL- HRUĠ TAL - LOTT B.
Name and address of the manufacturer responsible for batch release
Isem u indirizz tal- manifattur( i) responsabbli għall- ħruġ tal- lott.
Immeuble "Le Wilson" 70 avenue du Général de Gaulle F-92800 Puteaux France
Orphan Europe SARL Immeuble “ Le Wilson ” 70 avenue du Général de Gaulle F- 92800 Puteaux France
Medicinal product subject to restricted medical prescription (see annex I:
Prodott mediċinali jingħata b' riċetta ristretta tat- tabib (Ara Anness I:
Summary of Product Characteristics, section 4.2)
Sommarju tal - Karatteristiċi tal - Prodott, sezzjoni 4. 2).
The MAH must ensure that the system of pharmacovigilance is in place and functioning before the product is placed on the market and for as long as the marketed product remains in use.
Id- Detentur ta ’ l- Awtorizzazzjoni għat- Tqegħid fis- Suq għandu jiżgura li s- sistema ta ’ farmakoviġilanza tinsab f’ postha u qegħda taħdem qabel mal- prodott jitqiegħed fis- suq u sakemm il- prodott kumerċjalizzat jibqa ’ fis- suq.
The Marketing Authorisation Holder commits to performing the studies as detailed in the Pharmacovigilance Plan.
Id- Detentur ta ’ l- Awtorizzazzjoni għat- Tqegħid fis- Suq jimpenja ruħu li jwettaq l- istudji kif dettaljat fil- Pjan ta ’ Farmakoviġilanza.
10 PARTICULARS TO APPEAR ON THE OUTER PACKAGING
10 TAGĦRIF LI GĦANDU JIDHER FUQ IL- PAKKETT TA ’ BARRA
Cystadane 1 g oral powder Betaine anhydrous
Cystadane 1 g trab għall- ħalq Betaine anidru
Three measuring spoons dispense 1 g, 150 mg and 100 mg of betaine anhydrous.
Tliet kuċċarini bħala miżuri jagħtu 1 g, 150 mg u 100 mg ta ’ betaine anidru.
180 g of oral powder and three measuring spoons.
180 g ta 'trab għall- ħalq u tliet imgħaref tal- kejl.
Lightly shake the bottle before opening.
Jittieħed mill- ħalq.
Read the package leaflet before use.
Ħallat daqsxejn il- flixkun qabel ma tiftħu.
1 month.
Jiskadi {xahar/ sena} Wara li jinfetaħ l- ewwel darba: jiskadi fi żmien xahar.
Opened:
Miftuħ fil:
12 PARTICULARS TO APPEAR ON THE THE IMMEDIATE PACKAGING
12 TAGĦRIF LI GĦANDU JIDHER FUQ IL- PAKKETT LI JMISS MAL- PRODOTT
180 g of oral powder.
180 g ta 'trab għall- ħalq.
Lightly shake the bottle before opening.
Ħallat daqsxejn il- flixkun qabel ma tiftħu.
Read the package leaflet before use
Aqra l- fuljett ta 'tagħrif qabel l- użu.
1 month.
Jiskadi {xahar/ sena} Wara l- ewwel ftuħ: xahar.
SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF APPROPRIATE
PREKAWZJONIJIET SPEĊJALI GĦAR- RIMI TA ’ PRODOTTI MEDIĊINALI MHUX UŻATI JEW SKART MINN DAWN IL- PRODOTTI MEDIĊINALI, JEKK APPLIKABBLI
15 PACKAGE LEAFLET:
15 FULJETT TA ’ TAGĦRIF:
You may need to read it again.
Jista 'jkollok bżonn terġa ’ taqrah.
- If any of the side effects gets serious, or if you notice any side effects not listed in this leaflet,
- Jekk xi wieħed mill- effetti kollaterali jiggrava jew jekk tinnota xi effetti kollaterali li m’ humiex
What Cystadane is and what it is used for 2.
X’ inhu Cystadane u għalxiex jintuża 2.
Before you take Cystadane 3.
Qabel ma tieħu Cystadane 3.
How to take Cystadane 4.
Kif għandek tieħu Cystadane 4.
Possible side effects 5 How to store Cystadane 6.
Effetti kollaterali li jista ’ jkollu 5 Kif taħżen Cystadane 6.
WHAT CYSTADANE IS AND WHAT IT IS USED FOR
X’ INHU CYSTADANE U GĦALXIEX JINTUŻA
Cystadane contains betaine anhydrous which is intended to be an adjunctive treatment of homocystinuria, an inborn error of metabolism.
Cystadane fih betaine anidru li hu intiż biex ikun trattament aġġuntiv għall- omoċistinurja, difett li wieħed jitwieled biħ tal- metaboliżmu.
Methionine is an amino acid which is present in regular food protein (e. g. meat, fish, milk, cheese, eggs).
Methionine huwa aċidu amminiku li hu preżenti bħala proteina regolari ta 'l- ikel (e. g. laħam, ħut, ħalib, ġobon, bajd).
It is converted into homocysteine which is then normally converted into cysteine during digestion.
Huwa jiġi konvertit f' homocysteine li ġeneralment jiġi konvertit għal cysteine waqt id- diġestjoni.
Homocystinuria is a disease caused by the accumulation of homocysteine which is not converted to cysteine and is characterized by formation of clots in the veins, bone weakness, and skeletal and crystalline lens abnormalities.
L- omoċistinurja hija marda kaġunata mill- akkumulazzjoni ta 'homocysteine li ma jiġix konvertit f' cysteine u hu karaterizzat bil- formazzjoni ta' emboli fil- vini, dgħjufija fl- għadam, u anormalitajiet skeletriċi u tal- lenti kristallina.
The use of Cystadane together with other treatments such as vitamin B6, vitamin B12, folate and a specific diet aims to reduce the elevated homocysteine levels in your body.
L- użu ta 'Cystadane flimkien ma' mediċini oħra bħal vitamina B6, vitamina B12, folate u dieta speċifika għandha l- għan li tnaqqas il- livelli elevati ta 'omoċisteina fil- ġisem tiegħek.
BEFORE YOU TAKE CYSTADANE
QABEL MA TIEĦU CYSTADANE
Do not take Cystadane If you or your child are allergic (hypersensitive) to betaine.
Tieħux Cystadane Jekk inti jew it- tifel/ tifla tiegħek allerġiċi (tbati/ ibatu minn sensittività eċċessiva) għal betaine.
Take special care with Cystadane If you notice side effects like headaches, vomiting or a change in your vision, please contact your doctor immediately, they could be signs of a brain oedema.
Oqgħod attent ħafna b' Cystadane Jekk tinduna b' xi effetti kollaterali bħal uġigħat ta 'ras, remettar jew bidla fil- vista, jekk jogħġbok ikkuntattja lit- tabib tiegħek minnufih, billi jistgħu jkunu sinjali ta' edema fil- moħħ.
In that case your doctor will monitor your methionine level in your body and may review your diet.
Fi kwalunkwe każ it- tabib tiegħek ser jissorvelja l- livell ta 'methionine fil- ġisem tiegħek u jista' jirrivedi d- dieta tiegħek.
Your treatment with betaine may need to be interrupted.
Jista 'jkun li t- trattament tiegħek b' betaine għandha mnejn tiġi interrotta.
If you are treated with Cystadane and with an amino-acid mixture and if you need to take other medicines at the same time, leave 30 minutes between the intake (see “ taking other medicines”).
Jekk qed tiġi trattat b' Cystadane u b' taħlita ta 'aċidi amminiċi u jekk għandek bżonn tieħu mediċini oħra fl- istess żmien, ħalli 30 minuta bejn kull teħid (ara “ meta tieħu mediċini oħra).
Taking other medicines If you are taking amino-acid mixture or medicines such as vigabatrin or Gaba analogues, please tell your doctor as they might interact with your treatment with betaine.
Meta tieħu mediċini oħra Jekk qed tieħu taħlita ta ’ aċidi amminiċi bħal vigabatrin jew analogi Gaba, jekk jogħġbok għid lit - tabib tiegħek billi jistgħu jinħolqu reazzjonijiet mat- trattament tiegħek ma ’ betaine.
16 Pregnancy and breast-feeding Please inform your doctor if you are pregnant or breast-feeding.
16 Tqala u Treddigħ Jekk jogħġbok għarraf lit- tabib tiegħek jekk inti tqila jew jekk qed tredda '.
Your doctor will decide if the medicine may be used during pregnancy and breast-feeding.
It- tabib tiegħek ser jiddeċiedi jekk il- mediċina tistax tiġi użata waqt it- tqala jew it- treddigħ.
Driving and using machines No studies on the effects on the ability to drive and use machines have been performed.
Sewqan u tħaddim ta 'magni Ma sarux studji dwar l- effetti fuq il- ħila biex issuq jew tħaddem magni.
HOW TO TAKE CYSTADANE
KIF GĦANDEK TIEĦU CYSTADANE
Always take Cystadane exactly as your doctor has told you.
Dejjem għandek tieħu Cystadane skond il- parir tat- tabib.
You should check with your doctor or pharmacist if you are not sure.
Dejjem għandek taċċerta ruħek mat- tabib jew ma ’ l- ispiżjar tiegħek jekk ikollok xi dubju
The daily dose in adults, adolescents and children over 10 years of age is 6 g per day divided in 2 doses of 3 g per day.
Id- doża ta 'kuljum fl- adulti, l- adoloxxenti u t- tfal li għandhom aktar minn 10snin hi ta' 6 g kuljum li tiġi maqsuma f' 2 dożi ta '3 g kuljum.
In children less than 10 years of age, the daily dose will be calculated from the child’ s body weight by your doctor.
Fi tfal li għandhom inqas minn 10 snin, id- doża ta 'kuljum hi kalkulata skond il- piż korporali tat- tfal mit- tabib tiegħek.
You or your child will therefore need regular blood tests to determine the correct daily dose.
Għalhekk, int, jew it- tifel/ tifla tiegħek ikollkom bżonn tagħmlu testijiet regolari tad- demm biex tiġi stabbilita d- doża korretta.
The use of this medicine will be supervised by a physician experienced in the treatment of patients with homocystinuria.
L- użu ta 'din il- mediċina ser tkun taħt is- sorveljanza ta' tabib li għandu esperjenza fit- trattament ta 'pazjenti bl- omoċistinurja.
Cystadane should be taken orally (by mouth).
Cystadane għandu jittieħed oralment (mill- ħalq).
To measure the dose: • shake the bottle lightly before opening • take the correct measuring spoon:
Sabiex tkejjel id- doża: • ħallat daqsxejn il- flixkun qabel ma tiftħu. • ħu l- kuċċarina bil- miżura korretta:
the small spoon measures 100 mg of betaine anhydrous powder; the middle size spoon measures 150 mg of betaine anhydrous powder; the large spoon measures 1 g of betaine anhydrous powder.
il- kuċċarina ż- żgħira hija miżura għal 100 mg ta 'trab ta' betaine anidru; il- kuċċarina tan- nofs hija miżura għal 150 mg ta 'trab ta' betaine anidru; il- kuċċarina l- kbira hija miżura għal 1 mg ta 'trab ta' betaine anidru;
• pass the flat back of a knife over the top of the spoon • the powder left in the spoon is one spoonful • take the correct number of spoonfuls of powder from the bottle
• ħu kuċċarina mburġjata ta 'trab minn ġol- flixkun • għaddi l- parti ta' wara ta 'sikkina fuq il- parti ta' fuq tal- kuċċarina • it- trab li jitħalla fil- kuċċarina huwa kuċċarina waħda • ħu n- numru korrett ta 'kuċċarini ta' trab minn ġol- flixkun
Mix the measured dose of powder with water, juice, milk, formula or food until completely dissolved and ingest immediately after mixing.
Ħallat id- doża mkejla ta 'trab ma' l- ilma, il- meraq tal- frott, il- ħalib, it- trab tal- ħalib tat- trabi jew l- ikel sakemm jinħall kollu u ħudu immedjatament wara li tħallat.
Do not take a double dose to make up for forgotten doses.
Jekk tinsa tieħu Cystadane M’ għandekx tieħu doża doppja biex tpatti għal kull doża li tkun insejt tieħu.
POSSIBLE SIDE EFFECTS
EFFETTI KOLLATERALI LI JISTA ’ JKOLLU
Like all medicines, Cystadane can cause side effects, although not everybody gets them.
Bħal kull mediċina oħra, Cystadane jista ’ jkollu effetti kollaterali, għalkemm ma jidhrux fuq kulħadd.
The following side effects were reported as follows: very common (more than 1 out of 10 persons), common (more than 1 out of 100 persons and less than 1 out of 10 persons), uncommon (more than 1 out of 1,000 persons and less than 1 out of 100 persons), rare (more than 1 out of 10,000 persons and less than 1 out of 1,000 persons), very rare (less than 1 out of 10,000 persons).
L- effetti kollaterali li ġejjin ġew rapportati kif ġej: komuni ħafna (aktar minn 1 f' kull 10 persuni), komuni (aktar minn 1 f' kull 100 persuna imma inqas minn 1 f' kull 10 persuni), mhux komuni (aktar minn 1 f' kull 1, 000 persuna u inqas minn 1 f' 100 persuna), rari (aktar minn 1 f' 10, 000 persuna imma inqas minn 1 f' 1, 000 persuna), rari ħafna (inqas minn 1 f' 10, 000 persuna).
Very common: blood methionine increased.
Komuni ħafna: livell ogħla ta 'methionine fid- demm
17 Uncommon: decreased appetite, agitation, depression, irritability, personality disorder, sleep disturbance, oedema of the brain (see heading “Take special care with Cystadane” in section 2), dental disorders, diarrhoea, inflammation of the tongue, nausea, stomach discomfort, vomiting, hair loss, hives, skin odour abnormality and urinary incontinence.
17 Mhux komuni: tnaqqis fl- aptit, aġitazzjoni, depressjoni, irritabilità, disturbi fil- personalità, disturbi fl - irqadt, edema tal- moħħ (ara t- titlu “ Oqgħod attent ħafna b' Cystadane ” f' sezzjoni 2), disturbi dentali, dijaorrea, infjammazzjoni ta 'l - ilsien, nawsea, skonfort fl- istonku, remettar, telf ta' xahar, ħorriqija, riħa anormali tal- ġilda u inkontinenza awrinarja.
Since some of these side effects are serious, ask your doctor to explain their warning signs.
Imbilli xi ftit minn dawn l- effetti kollaterali huma serji, għid lit- tabib tiegħek jispjegalek dawn is - sinjali ta 'twissija.
If any of the side effects gets serious, or if you notice any other side effects not listed in this leaflet, please tell your doctor or pharmacist.
Jekk xi wieħed mill- effetti sekondarji jiggrava jew jekk tinnota xi effetti sekondarji li m’ humiex imsemmijin f’ dan il- fuljett, jekk jogħġbok, għid lit- tabib jew lill- ispiżjar tiegħek.
HOW TO STORE CYSTADANE
KIF TAĦŻEN CYSTADANE
Do not use Cystadane after the expiry date which is stated on the bottle label and the carton after EXP.
Tużax Cystadane wara d- data ta ’ skadenza li tidher fuq it- tikketta tal- flixkun u l- kartuna wara JIS.
The expiry date refers to the last day of that month.
Id - data ta ’ skadenza tirreferi għall- aħħar ġurnata ta ’ dak ix- xahar.
After the first opening of the bottle, the product should be used within 1 month.
Wara li tiftaħ il- flixkun l- ewwel darba, il- prodott għandu jintuża fi żmien xahar.
Medicine should not be disposed of via wastewater or household waste.
Il- mediċini m' għandhomx jintremew ma 'l- ilma tad- drenaġġ jew ma' l- iskart domestiku.
These measures will help protect the environment.
Dawn il- miżuri għandhom jgħinu għal protezzjoni ta 'l- ambjent.
What Cystadane contains
X' fih Cystadane
- The active substance is betaine anhydrous.
- Is- sustanza attiva hi betaine anidru.
1 g of oral powder contains 1 g of betaine anhydrous.
Tliet miżuri
Three measuring spoons dispense 1 g, 150 mg and 100 mg of betaine anhydrous.
jagħtu 1 g, 150 mg u 100 mg ta 'betaine anidru.
- There is no other ingredient.
- M' hemm l- ebda sustanza oħra.
What Cystadane looks like and contents of the pack Cystadane is a white crystalline oral powder.
Id- dehra ta 'Cystadane u d- daqs tal- pakkett Cystadane huwa trab kristallin abjad li jittieħed mill- ħalq.
It is presented in bottles with child resistant closures.
Jiġi fi fliexken b' għotjien li huma reżistenti għat- tfal.
Each bottle contains 180 g of betaine anhydrous powder.
Kull flixkun fih 180 g ta 'trab ta' betaine anidru.
Three measuring spoons are enclosed in the carton to assist you in measuring out the dose prescribed by your doctor.
Tliet imgħaref bħala miżuri jinsabu ġol kartuna sabiex jgħinuk tkejjel id- doża preskritta mit- tabib tiegħek.
Marketing Authorisation Holder and Manufacturer Orphan Europe SARL Immeuble “Le Wilson” F - 92058 Paris-La Défense France
Id- Detentur ta 'l- awtorizzazzjoni għat- tqegħid fis- Suq u l- Manifattur Orphan Europe SARL Immeuble “ Le Wilson ” F – 92058 Paris- La Défense Franza
18 For any information about this medicinal product, please contact the local representative of the Marketing Authorisation Holder.
18 Għal kull tagħrif dwar dan il- prodott mediċinali, jekk jogħġbok ikkuntattja lir- rappreżentant lokali tad - Detentur ta ’ l- Awtorizzazzjoni għat- Tqegħid fis- Suq.
It explains how the Committee for Medicinal products for Human Use (CHMP) assessed the studies performed, to reach their recommendations on how to use the medicine.
Huwa jispjega kif il- Kumitat għal Prodotti Mediċinali għall- Użu mill- Bniedem (CHMP) ivvaluta l- istudji mwettqa, biex jissodisfa r- rakkomandazzjonijiet tagħhom dwar kif tintuża l- mediċina.
What is CYSTAGON?
X’ inhu CYSTAGON?
CYSTAGON is a medicine containing the active ingredient cysteamine bitartrate (also known as mercaptamine bitartrate).
CYSTAGON hu mediċina li fiha l- ingredjent attiv cysteamine.
It is available as capsules (50 and 150 mg).
Jiġi bħala kapsuli (tal- 50 u tal - 150 mg).
What is CYSTAGON used for?
Għal x’ hiex jintuża CYSTAGON?
CYSTAGON is used to manage patients who have nephropathic (kidney) cystinosis.
CYSTAGON jintuża fil- ġestjoni ta ’ pazjenti li jkollhom nephropathic cystinosis.
Cystinosis is a rare inherited disorder where cystine builds up within body cells, preventing them from working correctly.
Cystinosis hi marda rari li tintiret li fiha, il- livelli ta ’ cystine fiċ- ċelloli tal- ġisem jiżdiedu u dan ma jħallihomx jaħdmu sew.
This leads to kidney problems and spreads to other parts of the body, including the eyes, some glands (thyroid, gonads, pancreas), the central nervous system, muscles and the liver.
Dan iwassal għal problemi fil- kliewi u tinfirex f’ partijiet oħra tal- ġisem, inklużi l- għajnejn, xi glandoli (it- tirojde, il- glandoli riproduttivi, il- frixa) is- sistema nervuża ċentrali, il- muskoli u l- fwied.
Stunted growth is a symptom of the condition.
It- twaqqif tat- tkabbir hu sintomu tal- kundizzjoni.
How is CYSTAGON used?
Kif jintuża CYSTAGON?
CYSTAGON treatment should be started by a doctor who has experience in the treatment of cystinosis.
It- trattament b’ CYSTAGON għandu jinbeda minn tabib li jkollu esperjenza fit- trattament tas - cystinosis.
Cystine levels in white blood cells should be monitored to adjust the dose.
Il- livelli ta ’ cystine fiċ- ċelloli tad- demm bojod għandhom jiġu sorveljati biex tkun aġġustata d- doża.
For children up to the age of 12 years, the recommended dose is 1.30 g/ m2/ day divided into 4 daily doses.
Għat- tfal ta ’ 12- il sena jew inqas, id- doża rakkomandata hi ta ’ 1. 30 g/ m2 kuljum maqsuma f’ 4 dożi.
For patients over the age of 12 and over 50 kg in weight, the recommended dose is 2 g/ day, divided into 4 daily doses.
Għal pazjenti ta ’ iktar minn 12- il sena u li jiżnu iktar minn 50 kg, id- doża rakkomandata hi ta ’ 2g kuljum, maqsuma f’ erba ’ dożi ndaqs.
Starting doses should be one quarter to one sixth of the final expected doses.
Id- dożi tal- bidu għandhom ikun bejn kwart u wieħed minn kull sitta tad- dożi finali mistennija.
The starting dose should be increased gradually over 4-6 weeks.
Id- dożi tal- bidu għandhom jiżdiedu gradwalment matul 4 sa 6 ġimgħat.
How does CYSTAGON work?
Kif jaħdem CYSTAGON?
CYSTAGON reacts with cystine to form cysteine and a cysteine-cysteamine salt (mixed disulfide).
CYSTAGON jirreaġixxi ma ’ cystine biex jifforma cysteine u melħ tas- cysteine- cysteamine (disulfide imħallat).
This salt is then removed from the cells by a system that transports another amino acid (lysine).
Dan il- melħ imbagħad jitneħħa miċ- ċelloli b’ sistema li tittrasporta amino aċid (lysine) ieħor.
The amount of cystine in the organs is reduced and this limits the damage to these organs.
L- ammont ta ’ cystine fl- organi jitnaqqas u dan jillimita l- ħsara f’ dawn l- organi.
How has CYSTAGON been studied?
Kif ġie studjat CYSTAGON?
CYSTAGON has been investigated in three clinical studies including 234 patients over a 12-year period.
CYSTAGON ġie investigat fi tliet studji kliniċi li fihom ħadu sehem 234 pazjent fuq perjodu ta ’ 12- il sena.
These studies included children and newly recruited patients in whom two different doses were tested.
F’ dawn l- istudji fost l- oħrajn ħadu sehem tfal u pazjenti reklutati riċentament li fihom ġew ittestjati żewġ dożi differenti.
Because the disease is very serious, it was not possible for ethical reasons to compare CYSTAGON directly to a dummy treatment.
Billi l- marda hi sejra ħafna, għal raġunijiet etiċi ma kienx possibbli li CYSTAGON jitqabbel direttament ma ’ trattament finta.
The comparison was done instead with a group of
Il- paragun minflok sar ma grupp ta ’ pazjenti
(44-20) 74 18 84 00 Fax (44-20) 74 18 84 16 E-mail: mail@emea. europa. eu http: / /www. emea. europa. eu ©EMEA 2007 Reproduction and/ or distribution of this document is authorised for non commercial purposes only provided the EMEA is acknowledged patients who had been treated with a dummy treatment as part of another, unrelated trial.
(44- 20) 74 18 84 00 Fax (44- 20) 74 18 84 16 E- mail: mail@ emea. europa. eu http: // www. emea. europa. eu ©EMEA 2007 Reproduction and/ or distribution of this document is authorised for non commercial purposes only provided the EMEA is acknowledged li kienu ġew trattati bi trattament finta bħala parti minn studju ieħor li m’ huwiex relatat.
The studies looked at the kidney function, the survival and the growth rate of the patients.
L- istudji ħarsu lejn il- funzjoni tal- kliewi, is- sopravivenza u r- rata ta ’ l- iżvilupp tal- pazjenti.
What benefit has CYSTAGON shown during the studies?
X’ inhuma l- benefiċċji ta ’ CYSTAGON li ntwerew f’ dawn l- istudji?
The three studies have shown that CYSTAGON delays kidney problems, and the need for dialysis or renal transplantation when treatment is started at an early age in patients with good kidney function.
It- tliet studji wrew li CYSTAGON itawwal iż- żmien qabel ma ’ jiżviluppaw problemi fil- kliewi, u l - ħtieġa ta ’ dijaliżi jew trapjant tal- kliewi meta t- trattament jinbeda minn età żgħira f’ pazjenti b’ funzjoni tajba tal- kliewi.
It also improves survival and growth rate in the children treated.
Itejjeb ukoll is- sopravivenza u r- rata ta ’ tkabbir ta ’ tfal li jkunu qed jiġu ttrattati.
What is the risk associated with CYSTAGON?
X’ inhu r- riskju assoċjat ma ’ CYSTAGON?
The side effects of CYSTAGON are mainly connected with the digestive system.
L- effetti sekondarji ta ’ CYSTAGON huma prinċiparjament marbuta mas- sistema diġestiva.
The most common side effects (seen in more than 1 patient in 10) are anorexia (loss of appetite), vomiting, nausea (feeling sick), diarrhoea, lethargy (lack of energy) and pyrexia (fever).
L- iktar effetti sekondarji komuni (li dehru f’ iktar minn pazjent wieħed minn kull għaxra) huma l- anoressija (nuqqas ta ’ aptit), id- diareja, il- letarġija (nuqqas ta ’ enerġija) u d- deni.
For the full list of all side effects reported with CYSTAGON, see the Package Leaflet.
Għal- lista sħiħa ta ’ l- effetti sekondarji rrappurtati b’ CYSTAGON, ara l- Fuljett ta ’ Tagħrif.
CYSTAGON should not be used in people who may be hypersensitive (allergic) to cysteamine or any of the other ingredients, or to penicillamine.
CYSTAGON m’ għandux jintuża minn persuni li huma allerġiċi għal cysteamine jew għal xi sustanzi oħra tiegħu, jew għal peniċillamine.
It should also not be used in women who are breast- feeding, or who are or may be pregnant (particularly during the first three months) unless it is clearly necessary.
Lanqas m’ għandu jintuża minn nisa li jkunu qed ireddgħu, jew li jkunu jew jistgħu joħorġu tqal (b’ mod partikolari fl- ewwel tliet xhur) sakemm dan ma jkunx neċessarju b’ mod ċar.
Why has CYSTAGON been approved?
Għaliex ġie approvat CYSTAGON?
Cystinosis is a rare, fatal disease, and CYSTAGON is considered a useful medicine for this disease, for which no alternative treatments are available.
Cystinosis hi marda rari u fatali u CYSTAGON hu meqjus mediċina utli għal din il- marda, li għaliha ma jeżisti l- ebda trattament alternattiv.
The Committee for Medicinal products for Human Use (CHMP) decided that CYSTAGON’ s benefits are greater than its risks and recommended that CYSTAGON be given marketing authorisation.
Il- Kumitat għall- prodotti Mediċinali għal Użu mill- Bniedem (CHMP) iddeċieda li CYSTAGON għandu iktar benefiċċji milli riskji u rrakkomanda li CYSTAGON jingħata awtorizzazzjoni għat- tqegħid fis- suq.
CYSTAGON was originally authorised under "Exceptional Circumstances", because, as the disease is rare, limited information was available at the time of approval.
CYSTAGON kien oriġinarjament awtorizzat taħt "Ċirkustanzi ta ’ Eċċezzjoni", għax, billi l- marda hi rari, it- tagħrif disponibbli fi żmien l- approvazzjoni kien limitat.
As the company had supplied the additional information requested, the "Exceptional Circumstances" ended on 17 April 2007.
Billi l- kumpanija kienet forniet it - tagħrif addizzjonali mitlub, iċ - "Ċirkustanzi ta ’ Eċċezzjoni" ntemmu fis- 17 ta ’ April 2007.
Other information about CYSTAGON:
Aktar tagħrif dwar CYSTAGON:
The European Commission granted a marketing authorisation valid throughout the European Union for CYSTAGON to Orphan Europe on 23 June 1997.
Il- Kummissjoni Ewropea tat awtorizzazzjoni għat- tqegħid fis- suq valida madwar l- Unjoni Ewropea kollha għal CYSTAGON lil Orphan Europe fit- 23 ta ’ Ġunju 1997.
The marketing authorisation was renewed on 23 June 2002 and on 23 June 2007.
L- awtorizzazzjoni għat- tqegħid fis - suq ġiet imġedda fit- 23 ta ’ Ġunju 2002 u fit- 23 ta ’ Ġunju 2007.
The full EPAR for CYSTAGON can be found here.
L- EPAR sħiħ għal CYSTAGON jista ’ jinstab hawnhekk.
This summary was last updated in 06-2007.
Dan is- sommarju ġie aġġornat f’ 06- 2007.
CYSTAGON 50 mg hard capsules
CYSTAGON 50 mg Kapsuli Iebsin
Each hard capsule contains 50 mg of cysteamine (as mercaptamine bitartrate).
Kull kapsula iebsa fiha 50 mg ta’ cysteamine bitartrate (bħala mercaptamine bitartrate),.
For a full list of excipients, see section 6.1.
Għal- lista sħiħa ta ’ sustanzi mhux attivi, ara sezzjoni 6. 1
Hard Capsule White, opaque hard capsules with CYSTA 50 on the body and MYLAN on the cap.
Kapsula Iebsa Kapsuli iebsin bojod, opaki b’ CYSTA 50 fuq il- korp u MYLAN fuq ir- ras.
4.1 Therapeutic indication
4. 1 Indikazzjonijiet terapewtiċi
CYSTAGON is indicated for the treatment of proven nephropathic cystinosis.
CYSTAGON hu indikat fit- trattament ta ’ ċistinożi nefropatika ppruvata.
Cysteamine reduces cystine accumulation in some cells (e. g. leukocytes, muscle and liver cells) of nephropathic cystinosis patients and, when treatment is started early, it delays the development of renal failure.
Cysteamine inaqqas l - akkumulazzjoni ta ’ cystine f’ xi ċelluli (eż. lewkoċiti, ċelloli tal- muskolu u tal- fwied) f’ pazjenti b’ ċistinożi nefropatika u, meta t- trattament jinbeda fi żmien bikri, dan idewwem l- iżvilupp ta ’ insuffiċjenza renali.
CYSTAGON treatment should be initiated under the supervision of a physician experienced in the treatment of cystinosis.
It- trattament b’ CYSTAGON għandu jinbeda taħt is- superviżjoni ta ’ tabib b’ esperjenza fit- trattament taċ- ċistinożi.
The goal of therapy is to keep leucocyte cystine levels below 1 nmol hemicystine/ mg protein.
L- iskop tat- terapija hu li l- livell ta’ cystine fil- lewkoċiti jinżamm taħt il- 1 nmol ta ’ hemicystine/ mg ta ’ proteina.
White blood cell (WBC) cystine levels should therefore be monitored to adjust the dose.
L- ammont ta ’ cystine fiċ- ċelloli bojod tad- demm għandu għalhekk jiġi osservat sabiex tkun tista ’ tiġi mibdula d- doża.
The WBC levels should be measured 5 to 6 hours after dosing and should be checked frequently when initiating therapy (e. g. monthly) and every 3-4 months when on a stable dose.
L- ammont ta 'ċelloli bojod tad- demm għandhu jiġi mkejjel kull 5 sa 6 sigħat wara l- għoti tad- doża u għandu jkun ikkontrollat regolarment wara l- bidu tat- terapija (eż. kull xahar) u kull 3- 4 xhur meta d- doża tkun stabbli.
• For children up to age 12 years, CYSTAGON dosing should be on the basis of body surface area (g/ m2/ day).
• Għat- tfal li għandhom sa 12- il sena, id- doża ta 'CYSTAGON għandha tingħata skond l- erja superfiċjali tal- ġisem (g/ m2/ kuljum).
The recommended dose is 1.30 g/ m2/ day of the free base divided four times daily. • For patients over age 12 and over 50 kg weight, the recommended CYSTAGON dose is 2 g/ day, divided four times daily.
Id- doża rrakkomandata hi ta '1. 30 g/ m2/ kuljum tal- bażi libera mqassma f' erba' darbiet kuljum. • Għal pazjenti li għandhom aktar minn 12- il sena u għandhom piż ta 'aktar minn 50 kg id- doża rrakkomandata ta' CYSTAGON hi ta '2 g/ kuljum, li titqassam f’ erba' darbiet kuljum.
Starting doses should be 1/ 4 to 1/ 6 of the expected maintenance dose, increased gradually over 4- 6 weeks to avoid intolerance.
Id- dożi inizjali għandhom ikunu ta ’ 1/ 4 sa 1/ 6 tad- doża ta ’ manteniment li mistennija tingħata, u tiżdied gradwalment fuq perijodu ta '4- 6 ġimgħat sabiex tiġi evitata l- intolleranza għall- prodott mediċinali.
The dose should be raised if there is adequate tolerance and the leucocyte cystine level remains > 1 nmol hemicystine/ mg protein.
Id- doża għandha tiżdied jekk it- tolleranza tkun adegwata u l- livell ta ’ cystine fil- lewkoċiti jibqa ’ > 1 nmol hemicystine/ mg ta ’ proteina.
The maximum dose of CYSTAGON used in clinical trials was 1.95 g/ m2/ day.
L- ogħla doża ta ’ CYSTAGON użata fi provi kliniċi kienet ta ’ 1. 95 g / m2/ jum.
The use of doses higher than 1.95 g/ m2/ day is not recommended (see section 4.4).
L- użu ta 'dożi ogħla minn 1. 95 g/ m2/ kuljum m' huwiex rakkomandat (ara sezzjoni 4. 4).
Digestive tolerance of cysteamine is improved when the medicinal product is taken just after or with food.
It- tolleranza diġestiva ta ’ cysteamine hi aħjar meta l- prodott mediċinali jittieħed ma 'l- ikel jew eżatt wara l- ikel.
In children who are at risk of aspiration, aged approximately 6 years and under, the hard capsules should be opened and the content sprinkled on food.
Fi tfal b’ riskju ta ’ aspirazzjoni, f’ età ta ’ madwar 6 snin jew anqas, il- kapsuli iebsin għandhom jinfetħu u l- kontenut tagħhom jitraxxax fuq l- ikel.
However, acidic drinks, e. g. orange juice, should generally be avoided as the powder tends not to mix well and may precipitate out.
L- esperjenza turi li ikel bħal ħalib, patata u prodotti oħrajn
2 Patients on dialysis or post-transplantation:
2 b’ bażi ta ’ lamtu huma xierqa biex jitħalltu ma ’ dan it- trab.
Experience has occasionally shown that some forms of cysteamine are less well tolerated (i. e. leading to more adverse events) when patients are on dialysis.
L- esperjenza turi li kultant ċertu forom ta ’ cysteamine huma ttollerati anqas min- normal (i. e. li jwassal għal aktar każijiet avversi) meta l- pazjenti għaddejjin minn dijaliżi.
A closer monitoring of the leucocyte cystine levels is recommended in these patients.
Hu rrakkomandat li f’ dawn il - pazjenti ssir osservazzjoni aktar mill- qrib ta 'livelli ta ’ cystine fil- lewkoċiti.
Patients with hepatic insufficiency:
Pazjenti b’ insuffiċjenza epatika:
Dose adjustment is not normally required; however, leucocyte cystine levels should be monitored.
Ġeneralment mhux meħtieġ tibdil fid- doża, iżda għandha ssir osservazzjoni tal- livelli ta ’ cystine fil - lewkoċiti.
Hypersensitivity to the active substance or to any of the excipients The use of CYSTAGON is contra-indicated during breast-feeding.
CYSTAGON huwa kontra- indikat jekk qed tredda ’.
CYSTAGON should not be used during pregnancy, particularly during the first trimester, unless clearly necessary (see section 4.6 and section 5.3 as it is teratogenic in animals.
CYSTAGON m’ għandux jittieħed waqt it- tqala, b' mod partikolari matul l- ewwel trimestru, sakemm ma jkunx meħtieġ bl- aktar mod evidenti (ara sezzjoni 4. 6 u sezzjoni 5. 3), minħabba li nstab li hu teratoġeniku fl- annimali.
CYSTAGON is contraindicated in patients who have developed hypersensitivity to penicillamine.
CYSTAGON huwa kontra- indikat f’ pazjenti li żviluppaw sensittività eċċessiva għal cysteamine jew peniċillamina.
CYSTAGON therapy must be initiated promptly after confirmation of the diagnosis of nephropathic cystinosis to achieve maximum benefit.
4. 4 Twissijiet speċjali u prekawzjonijiet għall- użu
Nephropathic cystinosis must have been diagnosed by both clinical signs and biochemical investigations (leucocyte cystine measurements).
Id- dijanjosi ta 'ċistonożi nefropatika trid tkun saret kemm permezz ta' sinjali kliniċi w investigazzjonijiet bijokemikali (għadd ta’ cystine fil- lewkoċiti).
A few cases of Ehlers-Danlos like syndrome on elbows have been reported in children treated with high doses of different cysteamine preparations (cysteamine chlorhydrate or cystamine or cysteamine bitartrate) mostly above the maximal dose 1.95 g/ m2/ day.
Xi ftit każijiet jixbhu lis- sindromu ta ’ Ehlers- Danlos fil- minkbejn ġew irrapurtati fi tfal ittrattati b’ dożi għoljin ta ’ cysteamine f’ medicini differenti (cysteamine chlorhydrate or cystamine or cysteamine bitartrate) li ħafna minnhom huma ’ l fuq mid- doża massima ta ’ 1. 95 g/ m2/ kuljum.
These skin lesions were associated with skin striae and bone lesions first seen during an X-ray examination.
Dawn il- leżjonijiet fil- ġilda huma assoċjati ma ’ strije fil- ġilda u leżjonijiet fil- għadam li ntwerew l- ewwel darba waqt eżamijiet tar- raġġi X.
It is therefore recommended to monitor regularly skin and to consider X-ray examinations of the bone as necessary.
Għalhekk huwa rrakkomandat li wieħed jimmonitorja regolarment il- ġilda u li jikkonsidra eżamijiet tar- raġġi X għall- għadam bħala neċessarji.
Self-examination of the skin by the patient or the parents should also be advised.
Eżami tal- ġilda mill- pazjent innifsu jew mill- ġenituri għandu jiġi rrakkomandat ukoll.
If any similar skin or bone abnormalities appear, it is recommended to decrease the dose of CYSTAGON.
Jekk jidhru xi anormalitajiet simili fil- ġilda jew fl- għadam, huwa rrakkomandat li titnaqqas id- doża ta ’ CYSTAGON.
The use of doses higher than 1.95g/ m2/ day is not recommended (see sections 4.2 and 4.8).
L- użu ta 'dożi ogħla minn 1. 95 g/ m2/ kuljum m' huwiex irrakkomandat (ara sezzjonijiet 4. 2 u 4. 8).
Monitoring of blood cell count is recommended on a regular basis.
Hu rrakkomandat li jkun hemm monitoraġġ ta 'l- għadd ta ’ ċelloli tad- demm fuq bażi regolari.
Oral cysteamine has not been shown to prevent eye deposition of cystine crystals.
Instab li cysteamine meħud mill- ħalq ma waqqafx id- depożitu ta ’ kristalli ta 'cystine fil- għajn.
Therefore, where cysteamine ophthalmic solution is used for that purpose, its usage should continue.
Għalhekk, fejn tintuża soluzzjoni għall- għajn għal dak l- iskop, l- użu tagħha għandu jitkompla.
In contrast to phosphocysteamine, CYSTAGON does not contain phosphate.
F’ kuntrast ma 'phosphocysteamine, CYSTAGON ma fihx fosfat.
Most patients will already be receiving phosphate supplements and the dose of these may need to be altered when CYSTAGON is substituted for phosphocysteamine.
Bosta pazjenti diġà se jkunu qed jingħataw fosfat żejjed u d- doża ta ’ dawn jista ’ jkollha tinbidel meta CYSTAGON jieħu post phosphocysteamine.
Intact CYSTAGON hard capsules should not be administered to children under the age of approximately 6 years due to risk of aspiration (see section 4.2).
Kapsuli iebsin ta ’ CYSTAGON sħaħ m’ għandhomx jiġu mogħtija lit- tfal ta ’ taħt l- età ta ’ madwar 6 snin minħabba r- riskju ta ’ aspirazzjoni (ara sezzjoni 4. 2).
No interaction studies have been performed CYSTAGON can be administered with electrolyte and mineral replacements necessary for management of the Fanconi syndrome as well as vitamin D and thyroid hormones.
CYSTAGON jista ’ jiġi mogħti ma ’ elektroliti u sustanzi li jingħataw bħala sostitut ta ’ minerali meħtieġa għall- immaniġġar tas- sindromu ta ’ Fanconi kif ukoll ma ’ vitamina D u ormoni ta 'tirojde.
Indomethacin and CYSTAGON have been used simultaneously in some patients.
Indomethacin u CYSTAGON intużaw flimkien f’ xi pazjenti.
In cases of patients with kidney transplants, anti-rejection treatments have been used with cysteamine.
F’ każi ta ’ pazjenti bi trapjant tal- kliewi, intużaw trattamenti kontra r- rifjut ma 'cysteamine.
3 4.6 Pregnancy and lactation
Studji fuq annimali wrew tossiċità riproduttiva, inkluż teratoġenesi (ara sezzjoni 5. 3).
The potential risk for humans is unknown.
Ir- riskju potenzjali fin- nies, mhux magħruf.
The effect on pregnancy of untreated cystinosis is also unknown.
Lanqas m’ hu magħruf l- effett fuq it- tqala ta ’ ċistonożi mhux ikkurata.
Therefore, CYSTAGON should not be used during pregnancy, particularly during the first trimester, unless clearly necessary.
Għalhekk, CYSTAGON m’ għandux jintuża waqt it- tqala, speċjalment matul l- ewwel trimestru, ħlief meta jkun hemm bżonn speċifiku.
If a pregnancy is diagnosed or planned, the treatment should be carefully reconsidered and the patient must be advised of the possible teratogenic risk of cysteamine.
Jekk tkun hemm dijanjosi ta 'tqala jew tqala ppjanata, it- trattament għandu jkun jerġa kkunsiderat b' reqqa u l- pazjent għandu jiġi infurmat dwar ir- riskju tal- possibbilta ’ teratoġenika ta 'cysteamine.
CYSTAGON excretion in human's milk is unknown.
Mhux magħruf jekk CYSTAGON jiġix eliminat fil- ħalib ta ’ l- omm.
However, due to the results of animal studies in breast-feeding mothers and neonates (see section 5.3), breast-feeding is contraindicated in women taking CYSTAGON.
Madankollu, minħabba r- riżultati ta ’ studji fuq annimali fuq ommijiet li qed ireddgħu u fuq frieħ li għadhom kif jitwieldu (ara sezzjoni 5. 3), it- treddigħ hu kontra- indikat f ''nisa li qed jieħdu CYSTAGON.
CYSTAGON has minor or moderate influence on the ability to drive and use machines CYSTAGON may cause drowsiness.
CYSTAGON jista ’ jikkawża n- ngħas.
When starting therapy, patients should not engage in potentially hazardous activities until the effects of the medicinal product on each individual are known.
Meta tinbeda t- terapija, il- pazjenti m’ għandhomx iwettqu attivitajiet li jistgħu jkunu perikolużi sakemm l- effetti tal- prodott mediċinali fuq kull individwu jkunu magħrufa.
Approximately 35% of patients can be expected to experience adverse reactions.
Madwar 35% tal- pazjenti jistgħu jistennew li ser ikollhom reazzjonijiet avversi.
These mainly involve the gastrointestinal and central nervous systems.
Dawn jinvolvu prinċipalment is- sistema gastro- intestinali u s- sistema nervuża ċentrali.
When these effects appear at the initiation of cysteamine therapy, temporary suspension and gradual reintroduction of treatment may be effective in improving tolerance.
Meta dawn l- effetti jfeġġu fl - istadju bikri tat- terapija b' cysteamine, is- sospensjoni temporanja u l- introduzzjoni gradwali mill- ġdid ta 'trattament jistgħu jkunu effettivi sabiex itejjbu t- tolleranza.
Reported adverse reactions are listed below, by system organ class and by frequency.
L- effetti ta 'reazzjonijiet avversi li kienu rrapportati huma mniżżla hawn isfel, skond is- sistema ta' klassifikazzjoni fuq l- organi u skond il- frekwenza.
Frequencies are defined as: very common (≥ 1/ 10), common (≥ 1/ 100 to < 1/ 10) and uncommon (≥ 1/ 1,000 to < 1/ 100).
Frekwenzi huma mfissra bħala: komuni ħafna (≥ 1/ 10), komuni (≥1/ 100 sa < 1/ 10) u mhux komuni (≥1/ 1, 000 sa < 1/ 100).
Within each frequency grouping, undesirable effects are presented in order of decreasing seriouness
Fi ħdan kull ragruppament ta ’ frekwenzi l- effetti mhux mixtieqa huma ppreżentati skond ordni dejjem tonqos fis- serjetà
Investigations Blood and lymphatic system disorders Nervous system disorders
Infezzjonijiet u infestazzjonijiet Komuni: testijiet tal- funzjoni tal- fwied, anormali Disturbi tad- demm u tas- sistema limfatika Mhux komuni:
Common:
Lewkopenja Disturbi tas- sistema nervuża Komuni:
Liver function tests abnormal Uncommon:
Uġigħ ta ’ ras, enċefalopatija Mhux komuni: ngħas, aċċessjonijiet Disturbi gastro- intestinali Komuni ħafna:
Leukopenia Common:
Komuni:
Headache, encephalopathy Uncommon:
Uġigħ addominali, nifs bir- riħa, dispepsja, gastroenterite Mhux komuni:
Somnolence, convulsions
Ulċera gastro- intestinali Disturbi marbuta mal- kliewi u s- sistema tal - awrina
Gastrointestinal disorders
Mhux
Very common:
komuni:
Gastrointestinal ulcer
Sindromu nefrotiku
Renal and urinary disorders Skin and subcutaneous tissue disorders
Disturbi tal- ġilda u tat- tessut subkutaneji Disturbi muskolo- skeletali u tat- tessut
Skin odour abnormal, rash Uncommon:
Riħa tal- ġilda, anormali, raxx Mhux komuni:
Hair colour changes, skin striae, skin fragility (molluscoid pseudotumor on elbows)
Ix- xagħar jibdel il- kulur, striji fil- ġilda, faġilità fil- ġilda (molluscoid pseudotumor fil - minkbejn)
Musculoskeletal and connective tissue disorders Metabolism and nutrition disorders
konnettiv Disturbi marbuta mal- metaboliżmu u
Joint hyperextension, leg pain, genu valgum, osteopenia, compression fracture, scoliosis.
Iperestensjoni tal- ġog, uġigħ fir- riġlejn, genu valgum, osteopinja, fratturi ta 'kompressjoni, skoljosi.
Anorexia
Anoreksja
General disorders and administration site conditions Immune system disorders Psychiatric disorders
jittieħed Disturbi marbuta mas- sistema immuni Disturbi psikjattriċi
Lethargy, pyrexia Common:
Letarġija, deni Komuni:
Asthenia Uncommon:
Astenja Mhux komuni:
Nervousness, hallucination
Reazzjoni anafilattika Mhux komuni: nervożità, alluċinazzjoni
4 Two cases of nephrotic syndrome have been reported within 6 months of starting therapy with progressive recovery after treatment discontinuation.
Żewġ każi ta ’ sindromu nefrotiku kienu rrapurtati sa 6 xhur mill- bidu ta 'terapija b’ fejqan progressiv wara t- twaqqif ta' trattament.
Histology showed a membranous glomerulonephritis of the renal allograft in one case and hypersensitivity interstitial nephritis in the other.
L- istoloġija wriet glomerulonefrite tal- membrana tar- renal allograft f’ każ wieħed, u sensittività eċċessiva ta ’ nefrite interstizjali f’ każ ieħor.
A few cases of Ehlers-Danlos like syndrome on elbows have been reported in children chronically treated with high doses of different cysteamine preparations (cysteamine chlorhydrate or cystamine or cysteamine bitartrate) mostly above the maximal dose 1.95 g/ m2/ day.
Xi ftit każijiet li jixbhu s- sindromu ta ’ Ehlers- Danlos fuq il- minkbejn ġew irraportati fi tfal ittrattati kronikament b’ dożi għoljin ta ’ mediċini b’ tipi differenti ta ’ cysteamine (cysteame chlorhydrate jew cystamine jew cysteamine bitartrate) li ħafna minnhom huma ’ l fuq mid- doża massima ta ’ 1. 95 g/ m2/ kuljum).
In some cases, these skin lesions were associated with skin striae and bone lesions first seen during an X-ray examination.
F' xi ftit każijiet, dawn il- leżjonijiet fil- ġilda kienu assoċjati ma ’ striji fil- ġilda u leżjonijiet fl- għadam li ntwerew l- ewwel darba waqt eżamijiet tar- raġġi X.
Bone disorders reported were genu valgum, leg pain and hyperextensive joints, osteopenia, compression fractures, and scoliosis.
Disturbi marbuti ma ’ l- għadam irrapportati kienu genu valgum, uġigħ fir- riġlejn u ġogi iperestensivi, osteopinja, fratturi ta ’ kompressjoni u skoljosi.
In the few cases where histopathological examination of the skin was performed, the results suggested angioendotheliomatosis.
Fil- ftit każi fejn sar eżami ħistopatoloġiku tal- ġilda, ir- riżultati kienu jissuġerixxu anġjo - endoteljomatosi.
One patient subsequently died of acute cerebral ischemia with marked vasculopathy.
F’ xi pazjenti, il- lezjonijiet fil- ġilda fuq il- minkbejn irrigressaw wara tnaqqis fid- doża ta ’ CYSTAGON.
In some patients, the skin lesions on elbows regressed after CYSTAGON dose reduction.
Hu maħsub li cysteamine jista 'jtellef li ssir il- ħolqa ta ’ katina tal- fibri tal- kollaġen (ara sezzjoni 4. 4).
An overdose of cysteamine may cause progressive lethargy.
Doża eċċessiva ta ’ cysteamine tista ’ tikkawża letarġija progressiva.
Should overdosage occur, the respiratory and cardiovascular systems should be supported appropriately.
F’ każ ta ’ doża eċċessiva għandu jingħata l- appoġġ meħtieġ lis- sistemi respiratorji u kardjovaskulari.
No specific antidote is known.
L- ebda antidotu speċifiku m’ huwa magħruf.
It is not known if cysteamine is removed by haemodialysis.
Mhux magħruf jekk cysteamine jistax jitneħħa permezz ta ’ dijaliżi tad- demm.
Alimentary tract and metabolism product, ATC code:
Prodott ta 'l- apparat alimentari u tal- metaboliżmu, Kodiċi ATC:
Normal individuals and heterozygous subjects for cystinosis have white cell cystine levels of < 0.2, and usually below 1 nmol hemicystine/ mg protein, respectively.
Individwi normali u pazjenti eterożigotici għal ċistinożi għandhom livelli ta ’ cystine fiċ- ċelluli bojod ta ’ < 0. 2, u ġeneralment anqas minn 1 nmol ta ’ hemicystine/ mg ta ’ proteina, rispettivament.
Individuals with nephropathic cystinosis have elevations of white cell cystine above 2 nmol hemicystine/ mg protein
5 Individwi b’ ċistinożi nefropatika jkollhom żjieda ta 'cystine fiċ- ċelluli bojod ta ’ aktar minn 2 nmol ta ’ hemicystine/ mg ta ’ proteina.
Cysteamine reacts with cystine to form the mixed disulfide of cysteamine and cysteine, and cysteine.
Cysteamine tirreaġixxi ma 'cystine biex tifforma disulfide ta' cysteamine u cysteine, u cysteine.
The mixed disulfide is then exported from the lysosomes by an intact lysine transport system.
Disulfide mħallta tiġi mbagħad esportata mill- liżożomi permezz ta 'sistema ta ’ trasport ta ’ lysine sħiħa.
The decrease in leucocyte cystine levels is correlated to the cysteamine plasma concentration over the six hours following the administration of CYSTAGON.
It- tnaqqis fl- għadd ta ’ cystine fil- lewkoċiti hu marbut mal- konċentrazzjoni ta ’ cysteamine fil- plażma matul is- sitt sigħat wara l- għoti ta ’ CYSTAGON.