natgillin/translations-raw huggingface dataset:
The safety and efficacy of Aldurazyme in patients older than 65 years have not been established and no dosage regimen can be recommended in these patients.
Varnost in učinkovitost zdravila Aldurazyme pri bolnikih, starejših od 65 let, ni ugotovljena, zato pri teh bolnikih ne moremo priporočati režima odmerjanja.
The safety and efficacy of Aldurazyme in patients with renal or hepatic insufficiency have not been evaluated and no dosage regimen can be recommended in these patients.
Varnost in učinkovitost zdravila Aldurazyme pri bolnikih z jetrno ali ledvično okvaro ni ocenjena, zato pri teh bolnikih ne moremo priporočati režima odmerjanja
Severe hypersensitivity (e. g. anaphylactic reaction) to the active substance or to any of the excipients (see sections 4.4 and 4.8).
4. 4 Posebna opozorila in previdnostni ukrepi
4.4 Special warnings and precautions for use
Pri bolnikih, ki se zdravijo z zdravilom Aldurazyme, se lahko razvijejo reakcije ob infuziji.
Patients treated with Aldurazyme may develop infusion-associated reactions (IARs), defined as any related adverse event occurring during the infusion or until the end of the infusion day (see section 4.8).
To so kakršnikoli z infuzijo povezani neželeni dogodki, ki se pojavijo med infundiranjem ali do konca dneva, ko je bolnik prejel infuzijo (glejte poglavje 4. 8).
Severe infusion associated reactions have been reported in patients with pre-existent severe underlying upper airway involvement and therefore specifically these patients should continue to be closely monitored and only be infused with Aldurazyme in an appropriate clinical setting where resuscitation equipment to manage medical emergencies would be readily available.
Hude reakcije so se ob infuziji pojavile pri bolnikih, ki so imeli že prej močno prizadeta zgornja dihala, zato je treba te bolnike nenehno skrbno opazovati, zdravilo Aldurazyme pa se jim lahko infundira le v ustrezni klinični ustanovi, kjer imajo pri roki opremo za oživljanje za reševanje nujnih zdravstvenih primerov.
Careful consideration should be given to the patient’ s clinical status prior to administration of Aldurazyme.
Bolniki s predhodnimi akutnimi obolenji so še bolj izpostavljeni reakcijam ob infuziji zdravila Aldurazyme, zato je pri takih bolnikih potrebno nameniti dodatno pozornost na klinično stanje bolnika pred infundiranjem zdravila Aldurazyme.
Patients who have developed antibodies or symptoms of IARs should be treated with caution when administering Aldurazyme (see sections 4.3 and 4.8).
Aldurazyme uporabljajte previdno pri bolnikih, ki imajo protitelesa ali znake reakcij ob infuziji (glejte poglavje 4. 3 in 4. 8).
In clinical studies IARs were usually manageable by slowing the rate of infusion and by (pre-) treating the patient with antihistamines and/ or antipyretics (paracetamol or ibuprofen), thus enabling the patient to continue treatment.
V kliničnih študijah so reakcije ob infuziji navadno uspeli ublažiti s počasnejšim infundiranjem in (predhodnim) zdravljenjem z antihistaminiki in/ ali antipiretiki (paracetamolom ali ibuprofenom), tako da je bolnik lahko nadaljeval zdravljenje.
With initial administration of Aldurazyme or upon re-administration following interruption of treatment, it is recommended that patients be administered pretreatment medicines(antihistamines and/ or antipyretics) approximately 60 minutes prior to the start of the infusion, to minimise the potential occurrence of IARs.
Ob začetku uporabe zdravila Aldurazyme ali ob ponovni uporabi po prekinitvi zdravljenja priporočamo predhodno zdravljenje bolnika z antihistaminiki in/ ali antipiretiki (približno 60 minut pred infuzijo), kar bo zmanjšalo možnosti pojava reakcij ob infuziji.
If clinically indicated, administration of pretreatment medications with subsequent infusions of Aldurazyme should be considered.
Če je to klinično indicirano, premislite o predhodni terapiji z zdravili in poznejšimi infuzijami zdravila Aldurazyme.
In case of a mild or moderate IAR, treatment with antihistamines and paracetamol/ ibuprofen should be considered and/ or a reduction in the infusion rate to half the infusion rate at which the reaction occurred.
Ob pojavu blage ali zmerne reakcije ob infuziji poskusite zdraviti bolnika z antihistaminiki in paracetamolom ali ibuprofenom in/ ali za polovico zmanjšati hitrost infundiranja, ob kateri je prišlo do reakcije.
In case of a single severe IAR, the infusion should be stopped until the symptoms are resolved and treatment with antihistamines and paracetamol/ ibuprofen should be considered.
Če se pojavi enkratna težka reakcija ob infuziji, morate zdravljenje z infuzijo prekiniti, dokler so simptomi prisotni, in razmisliti o zdravljenju z antihistaminiki ter s paracetamolom ali ibuprofenom.
The infusion can be restarted with a reduction of the infusion rate to 1/ 2 – 1/ 4 the rate of the infusion at which the reaction occurred.
V nadaljevanju lahko dajete bolniku infuzijo s hitrostjo, ki je za četrtino do polovico manjša od hitrosti, ob kateri je prišlo do reakcije.
In case of a recurrent moderate IAR or re-challenge after a single severe IAR, pretreatment should be considered (antihistamines and paracetamol/ ibuprofen and/ or corticosteroids) and a reduction of the infusion rate to 1/ 2 – 1/ 4 the rate of the infusion at which the previous reaction occurred.
V primeru ponavljajočih se zmernih reakcij ali ponovni nevarnosti po enkratni težki reakciji ob infuziji, bo morda potrebno predhodno zdravljenje (z antihistaminiki, paracetamolom ali ibuprofenom in/ ali kortikosteroidi) in zmanjšanje hitrosti infundiranja na četrtino do polovico hitrosti, pri kateri se je pojavila prejšnja reakcija.
If these reactions occur, immediate discontinuation of Aldurazyme is recommended and appropriate medical treatment should be initiated.
3 Če pride do teh reakcij, priporočamo takojšnjo prekinitev zdravljenja z zdravilom Aldurazyme in ustrezne zdravstvene ukrepe.
3 This medicinal product contains sodium and is administered in 0.9% sodium chloride intravenous solution (see section 6.6).
To zdravilo vsebuje natrij; zdravilo infundirajte v obliki 0, 9% intravenske raztopine natrijevega klorida (glejte poglavje 6. 6).
The potential risk for humans is unknown.
Študije na živalih ne kažejo na neposredne ali posredne škodljive učinke na nosečnost, razvoj zarodka/ plodu, porod ali postnatalni razvoj (glejte poglavje 5. 3.) Možno tveganje za ljudi ni znano.
The majority of the related adverse events in the clinical trials were classified as infusion-associated reactions (IARs), experienced by 53% of the patients in the Phase 3 study (treated for up to 4 years) and 35% of the patients in the under 5 study (up to 1 year of treatment).
Večino z zdravilom povezanih neželenih dogodkov v kliničnih preskušanjih so opredelili kot reakcije povezane z infuzijami (infusion associated reactions, IARs)), do katerih je prišlo pri 53% bolnikov v tretji fazi študije (zdravljenje do 4 let) in 35% bolnikov mlajših od 5 let (zdravljenje do 1 leta).
The majority of the IARs were mild or moderate in intensity.
Večina z infuzijo povezanih reakcij je bila blagih ali zmernih.
The most frequent adverse drug reactions (ADRs) were: headache, nausea, abdominal pain, rash, arthralgia, backpain, pain at extremity, flushing, pyrexia, infusion site reactions, blood pressure increased, oxygen saturation decreased tachycardia and chills.
Najpogostejši neželeni učinki zdravila so bili: glavobol, navzea, bolečine v trebuhu, izpuščaji, artlargija, bolečine v hrbtu, bolečine v udih, navali vročine, pireksija, reakcije na mestih infuzije, zvišani krvni tlak, zmanjšana nasičenost s kisikom, tahikardija in mrzlica.
ADRs to Aldurazyme reported during the Phase 3 study and its extension in a total of 45 patients age 5 years and older and treated up to 4 years are listed below using the following categories of frequency: very common (≥ 1/ 10); common (≥ 1/ 100 to < 1/ 10).
Neželeni učinki zdravila Aldurazyme, o katerih so poročali v tretji fazi študije in njenem podaljšanju pri 45 bolnikih, starih 5 let in več ter zdravljenih do 4 leta, so razporejeni v spodnji preglednici po naslednjih kategorijah pogostosti: zelo pogosto (≥ 1/ 10); pogosto (≥ 1/ 100 do < 1/ 10).
MedDRA MedDRA
razred organskega sistema (po MedDRA)
System Organ Class
prednostni izraz (po MedDRA)
Preferred Term
pogostost
body temperature increased,
zvišana telesna temperatura,
Investigations
preiskave
common
pogosto
Cardiac disorders
srčne bolezni
Nervous system disorders
bolezni živčevja
very common
zelo pogosto
paraesthesia, dizziness,
parestezija, omotica
Respiratory, thoracic and mediastinal
bolezni dihal, prsnega koša in
disorders
mediastinalnega prostora
respiratory distress, dyspnoea, cough
težave z dihanjem, dispneja, kašelj
Gastrointestinal disorders
bolezni prebavil
nausea, abdominal pain
navzea, bolečine v trebuhu
vomiting, diarrhoea rash
bruhanje, driska izpuščaji angionevrotični edem, otekanje
common very common
pogosto zelo pogosto
Skin and subcutaneous tissue disorders angioneurotic edema, swelling face,
bolezni kože in podkožja
urticaria, pruritus, cold sweat, alopecia, hyperhidrosis arthropathy, arthralgia, back pain,
obraza, urtikarija, pruritus, hladno znojenje, alopecija, hiperhidroza artropatija, artlargija, bolečine v
musculoskeletal pain flushing Vascular disorders hypotension, pallor, peripheral coldness
bolezni mišično- skeletnega sistema in vezivnega tkiva
common very common common
hrbtu, bolečine v okončinah
pyrexia, Infusion site reaction
bolečine v mišicah in kosteh
very common
pogosto
General disorders and administration site
zelo pogosto navali vročine žilne bolezni pogosto hipotenzija, bledica, mrzle okončine zelo pogosto pireksija, reakcija na mestu infuzije
conditions
splošne težave in spremembe na mestu aplikacije
chills, feeling hot, feeling cold, fatigue, influenza like illness
mrzlica, občutek vročine, občutek mraza, utrujenost, gripi podobna bolezen
system disorders
bolezni imunskega sistema psihiatrične motnje
Psychiatric disorders
anafilaktična reakcija nemirnost
common
pogosto pogosto
Most of the ADRs were mild to moderate in severity.
Večina neželenih učinkov je bila blagih do zmernih glede resnosti.
Post-marketing experience of infusion associated reactions revealed reporting of pyrexia, chills and vomiting, mostly mild to moderate in intensity.
V obdobju po začetku prodaje zdravila so pri infundiranju zaznali redne pojave zvišane telesne temperature, mrzlice in bruhanja, povečini v blagi do zmerni obliki.
Additionally, a few patients who had a prior history of severe MPS I- related upper airway and pulmonary involvement, experienced severe reactions including bronchospasm, respiratory arrest, and facial oedema (see section 4.4).
Poleg tega so se pri nekaj bolnikih, ki so imeli zaradi MPS I hudo prizadeta zgornja dihala in pljuča, pojavile hude reakcije, vključno z bronhospazmom, zastankom dihanja in obraznim edemom (oglejte si odstavek 4. 4).
Paediatric population ADRs to Aldurazyme reported during a Phase 2 study in a total of 20 patients, under 5 years of age and mainly of the severe phenotype, treated up to 12 months are listed below.
Pediatrična skupina Neželeni učinki zdravila Aldurazyme, zabeleženi v drugi fazi študije pri 20 bolnikih, mlajših od 5 let in v glavnem s hujšo obliko fenotipa, zdravljenih do 12 mesecev, so navedeni v spodnji preglednici.
MedDRA
organski sistem (po MedDRA)
MedDRA
prednostni izraz (po MedDRA)
Frequency
pogostost
System Organ Class
preiskave
Preferred term
povišan krvni pritisk
Investigations
zelo pogosto
blood pressure increased
zmanjšana nasičenost s kisikom
saturation decreased tachycardia
tahikardija pireksija
Very common Very common
zelo pogosto zelo pogosto
General disorders and administration site conditions
splošne težave in spremembe na
pyrexia chills
mestu aplikacije
Very common Very common
zelo pogosto
5 In a phase 4 study 33 MPS1 patients received 1 of 4 dose regimens:
5 V 4. fazi študije je 33 bolnikov z MPS1 prejemalo 1 izmed 4 režimov odmerjanja:
100 U/ Kg IV every week (recommended dose), 200 U/ Kg IV every week, 200 U/ Kg IV every 2 weeks or 300 U/ Kg IV every 2 weeks.
100 e/ kg i. v. vsak teden (priporočeni odmerek), 200 e/ kg i. v. vsak teden, 200 e/ kg i. v. vsaka 2 tedna ali 300 e/ kg i. v. vsaka 2 tedna.
Immunogenicity Almost all patients developed IgG antibodies to laronidase.
Imunogenost Pri skoraj vseh bolnikih je prišlo do tvorbe protiteles IgG na laronidazo.
By the end of the Phase 3 study (or at time of early study withdrawal), 13/ 45 patients had no detectable antibodies by radioimmunoprecipitation (RIP) assay, including 3 patients that had never seroconverted.
Do zaključka tretje faze študije (ali ob času zgodnjega umika iz študije) pri 13 bolnikih (od 45 bolnikov) radioimunološki obarjalni preskus (RIP) ni zaznal protiteles, vključno pri 3 bolnikih, pri katerih ni nikoli prišlo do serokonverzije.
In addition 60 patients in the Phase 2 and 3 studies were tested for in-vitro neutralising effects.
Poleg tega so bili pri 60 bolnikih v drugi in tretji fazi študije opravljeni preizkusi za in- vitro nevtralizacijske učinke.
Four patients (three in the Phase 3 study and one in the Phase 2 study) showed marginal to low level in vitro inhibition of laronidase enzymatic activity, which did not appear to impact clinical efficacy and/ or urinary GAG reduction.
Pri štirih bolnikih (treh iz tretje faze študije in enem iz druge faze študije) je bila opažena mejna do nizka in vitro inhibicija encimske aktivnosti laronidaze, ki ni imela očitnega vpliva na klinično učinkovitost in/ ali zmanjšanje vrednosti GAG v urinu.
Pharmacotherapeutic group:
Farmakoterapevtska skupina: encimi.
Enzymes, ATC code:
Oznaka ATC:
MPS I is a heterogeneous and multisystemic disorder characterised by the deficiency of α -L-iduronidase, a lysosomal hydrolase which catalyses the hydrolysis of terminal α -L-iduronic residues of dermatan sulfate and heparan sulfate.
Mukopolisaharidoza tipa I je heterogena in multisistemska motnja, za katero je značilno pomanjkanje α- L- iduronidaze, lizosomske hidrolaze, ki katalizira hidrolizo α- L- iduronskih ostankov dermatan sulfata in heparan sulfata.
Reduced or absent α -L-iduronidase activity results in the accumulation of the GAGs, dermatan sulfate and heparan sulfate in many cell types and tissues.
Zmanjšano delovanje ali odsotnost α- L- iduronidaze povzroča kopičenje glikozaminoglikanov, dermatan sulfata in heparan sulfata v mnogih vrstah celic in tkiv.
After intravenous infusion, laronidase is rapidly removed from the circulation and taken up by cells into lysosomes, most likely via mannose-6 phosphate receptors.
Po intravenski infuziji laronidaza hitro zapusti krvni obtok in celice jo zajamejo v lizosome, najverjetneje z receptorji za manozo- 6- fosfat.
Purified laronidase is a glycoprotein with a molecular weight of approximately 83 kD.
Očiščena laronidaza je glikoprotein z molekularno maso približno 83 kD.
Laronidase is comprised of 628 amino acids after cleavage of the N-terminus.
Laronidaza je po odcepitvi N- terminala sestavljena iz 628 aminokislin.
Three clinical trials were performed with Aldurazyme to assess its efficacy and safety.
6 Z zdravilom Aldurazyme so bila opravljena tri klinična preskušanja za ocenitev njegove učinkovitosti in varnosti.
One study mainly assessed the safety and
Ena klinična študija je bila osredotočena predvsem na ocenjevanje učinka zdravila Aldurazyme na sistemske znake mukopolisaharidoze I, kot so slaba vzdržljivost,, restriktivna pljučna bolezen, obstrukcija zgornjih dihalnih poti, zmanjšan obseg gibanja sklepov, hepatomegalija in poslabšanje vida.
6 pharmacokinetics of Aldurazyme in patients less than 5 years old, but some efficacy measurements were included as well.
V eni študiji so ocenjevali predvsem varnost in farmakokinetiko zdravila Aldurazyme pri bolnikih, mlajših od 5 let, vključenih pa je bilo tudi nekaj meritev učinkovitosti.
Patients were enrolled with a Forced Vital Capacity (FVC) less than 80% of the predicted value and had to be able to stand for 6 minutes and to walk 5 meters.
Sodelovali so bolniki s forsirano vitalno kapaciteto (FVC) manj kot 80% od predvidene vrednosti ter so bili zmožni stati 6 minut in prehoditi 5 metrov.
Patients received either 100 U/ kg of Aldurazyme or placebo every week for a total of 26 weeks.
Študija je trajala 26 tednov in bolniki so vsak teden prejeli 100 e/ kg zdravila Aldurazyme ali placeba.
The primary efficacy endpoints were changes in percent of predicted normal FVC and absolute distance travelled in the six-minute walk test (6MWT).
Osnovna merila učinkovitosti so bile spremembe v odstotku predvidene normalne forsirane vitalne kapacitete in absolutne razdalje, ki so jo bili zmožni prehoditi v 6- minutnem preizkusu (6MWT).
All patients subsequently enrolled in an open label extension study where they all received 100 U/ kg of Aldurazyme every week for an additional 3.5 years (182 weeks).
Pozneje so vsi bolniki sodelovali v razširjeni odprti študiji, kjer so vsi vsak teden dobivali 100 e/ kg zdravila Aldurazyme dodatnih 3, 5 let (182 tednov).
p value
vrednost p
Confidence interval (95%)
Interval zaupanja (95%)
Percent Predicted
predvideni odstotek FVC
FVC (percentage point)
(odstotna točka) 6MWT (metri)
median
mediana srednja vrednost
0.009
3, 0 38, 1
0.9 - 8.6
0, 009 -
(meters)
0, 9 – 8, 6
median
mediana
The open label extension study showed improvement and/ or maintenance of these effects up to 208 weeks in the Aldurazyme/ Aldurazyme group and 182 weeks in the Placebo/ Aldurazyme group as indicated in the table below.
Razširjena odprta študija je pokazala izboljšanje in/ ali vzdrževanje navedenih učinkov do 208 tedna pri skupini, ki je prejemala Aldurazyme/ Aldurazyme, in 182 tednov pri skupini, ki je prejemala placebo/ Aldurazyme, kot je navedeno spodaj.
Aldurazyme/ Aldurazyme At 208 weeks
Aldurazyme/ Aldurazyme po 208 tednih
Placebo/ Aldurazyme At 182 weeks
Placebo/ Aldurazyme po 182 tednih
Percent predicted FVC (%)1 6 MWT (meters) Apnea/ Hypopnea Index (AHI) Shoulder flexion Range Of Motion (degrees) CHAQ/ HAQ Disability Index 2
predvideni odstotek FVC (%) 1 6 MWT (metri) Indeks apneja/ hipopneja (IAH) obseg gibanja ramena (stopinje) CHAQ/ HAQ indeks invalidnosti2
The decrease in percent predicted FVC is not clinically significant over this timeframe, and absolute lung volumes continued to increase commensurate with changes in height in growing paediatric patients.
Znižanje predvidenega odstotka FVC ni klinično pomembno v tem časovnem okviru, celotni volumen pljuč pa se je še naprej zviševal sorazmerno s spremembami v višini pediatričnih bolnikov, ki še rastejo.
2 Both groups exceeded the minimal clinically important difference (-0.24)
2 Obe skupini sta prekoračili najnižjo klinično pomembno razliko (- 0, 24)
Of the 26 patients with abnormal liver volumes at pre-treatment baseline, 22 (85%) achieved a normal liver size by the end of the study.
Izmed 26 bolnikov z abnormalnimi volumni jeter glede na izhodiščno točko, jih je 22 (85%) doseglo normalno velikost jeter do zaključka študije.
There was a rapid reduction in the excretion of urinary GAG (µg/ mg creatinine) within the first 4 weeks, which was maintained through the remainder of the study.
Hitro znižanje v izločanju GAG v urinu (µg/ mg
Urinary GAG levels decreased by 77% and 66% in the Placebo/ Aldurazyme and Aldurazyme/ Aldurazyme groups, respectively; at the end of the study one-third of the patients (15 of 45) had reached normal urinary GAG levels.
Vrednosti GAG v urinu so se znižale za 77% pri placebo/ Aldurazyme skupini oziroma 66% pri Aldurazyme/ Aldurazyme skupini.
7 To address the heterogeneity in disease manifestation across patients, using a composite endpoint that summed up clinically significant changes across five efficacy variables (percent predicted normal FVC, 6MWT distance, shoulder flexion range of motion, AHI, and visual acuity) the global response was an improvement in 26 patients (58%), no change in 10 patients (22%), and a deterioration in 9 patients (20%).
Heterogenost manifestacije bolezni pri bolnikih smo obravnavali z uporabo sestavljene končne vrednosti, ki je povzela klinično pomembne spremembe petih spremenljivk učinkovitosti (predvideni normalni odstotek FVC, razdalja 6 min preizkusa hoje (MWT), obseg gibanja ramena, IAH in ostrina vida); globalni odgovor je bil izboljšanje pri 26 bolnikih (58%), brez spremembe pri 10 bolnikih (22%) in poslabšanje pri 9 bolnikih (20%).
The patients were scheduled to receive Aldurazyme 100 U/ kg weekly infusions for a total duration of 52 weeks.
Bolniki so 52 tednov tedensko prejemali infuzije zdravila Aldurazyme po 100 e/ kg.
Four patients underwent dosage increases to 200 U/ kg for the last 26 weeks because of elevated urinary GAG levels at Week 22.
Pri štirih bolnikih se je zadnjih 26 tednov študije odmerek povišal na 200 e/ kg zaradi zvišanih vrednosti GAG v urinu v 22. tednu.
The mean urinary GAG level declined by 50% at Week 13 and was reduced by 61% at the end of the study.
Srednja vrednost GAG v urinu se je znižala za 50% v 13. tednu in za 61% ob zaključku študije.
Upon study completion, all patients showed reductions in liver size and 50% (9/ 18) had normal liver size.
Po zaključku študije je bilo pri vseh bolnikih opazno zmanjšanje velikosti jeter in 50% (9/ 18) je imelo normalno velika jetra.
The proportion of patients with mild left ventricular hypertrophy decreased from 53% (10/ 19) to 17% (3/ 18), and mean left ventricular mass normalized for body surface area decreased by 0.9 Z-Score (n=17).
Odstotek bolnikov z blago hipertropijo na levem prekatu se je znižal s 53% (10/ 19) na 17% (3/ 18), srednja masa levega prekata se je normalizirala, saj se je telesna površina znižala za 0, 9 Z- Score (n=17).
The younger patients with the severe phenotype (< 2.5 years) and all 4 patients with the intermediate phenotype exhibited a normal rate of mental development, whereas the older patients with a severe phenotype made limited or no gains in cognition.
Mlajši bolniki z resnim fenotipom (< 2, 5 let) in vsi 4 bolniki z vmesnim fenotipom so pokazali normalno stopnjo mentalnega razvoja, medtem ko so starejši bolniki z resnim fenotipom kazali omejen napredek ali nobenega napredka v kogniciji.
In this 26-week open label study, 33 MPS1 patients received 1 of 4 dose regimens of Aldurazyme:
V tej 26- tedenski odprti študiji je 33 bolnikov z MPS1 prejemalo 1 izmed 4 režimov odmerjanja:
100 U/ Kg IV every week (recommended dose), 200 U/ Kg IV every week, 200 U/ Kg IV every 2 weeks; or 300 U/ KgIV every 2 weeks.
100 e/ kg i. v. vsak teden (priporočeni odmerek), 200 e/ kg i. v. vsak teden, 200 e/ kg i. v. vsaka 2 tedna ali 300 e/ kg i. v. vsaka 2 tedna.
The 200 U/ Kg IV every 2 weeks regimen may be an acceptable alternative for patients with difficulty receiving weekly infusions; however, there is no evidence that the long term clinical efficacy of these two dose regimens is equivalent.
Režim z odmerkom 200 e/ kg i. v. vsaka 2 tedna je sprejemljivi alternativni režim za bolnike, ki imajo težave pri tedenskih infuzijah, vendar pa ni dokazov o ekvivalentnosti dolgotrajnejše klinične učinkovitosti teh dveh režimov odmerjanja.
This medicinal product has been authorised under “ Exceptional Circumstances”.
Zdravilo je pridobilo dovoljenje za promet v “ izjemnih okoliščinah ”.
The European Medicines Agency (EMEA) will review any new information which may become available every year and this SPC will be updated as necessary.
Evropska agencija za zdravila (EMEA) bo vsako leto ponovno pregledala vse nove podatke, ki bodo na voljo in posodobila povzetek glavnih značilnosti zdravila, če bo potrebno.
After intravenous administration of laronidase with an infusion time of 240 minutes and at a dose of 100 U/ kg body weight pharmacokinetic properties were measured at Weeks 1, 12 and 26.
Po intravenski uporabi laronidaze s časom infundiranja 240 minut in z odmerkom 100 e/ kg telesne mase so merili farmakokinetične lastnosti v 1., 12. in 26. tednu zdravljenja.
Mean ± SD
Srednja vrednost ± SD 0, 197 ± 0, 052
Mean ± SD
Srednja vrednost ± SD 0, 210 ± 0, 079
Mean ± SD
Srednja vrednost ± SD 0, 302 ± 0, 089
Cmax showed an increase over time.
8 Vrednost Cmax je sčasoma naraščala.