natgillin/translations-raw huggingface dataset:
A phase III trial (study 478) compared oral topotecan plus Best Supportive Care (BSC) (n = 71) with BSC alone (n = 70) in patients who had relapsed following first line therapy (median time to progression [TTP] from first-line therapy: 84 days for oral topotecan + BSC, 90 days for BSC) and for whom retreatment with intravenous chemotherapy was not considered appropriate.
Raziskava je bila opravljena pri bolnikih, pri katerih je po zdravljenju s terapijo prve izbire prišlo do relapsa (srednji čas do progresije, ki je minil od zdravljenja s terapijo prve izbire: 84 dni za zdravljenje s peroralnim topotekanom v kombinaciji z NPO; 90 dni za zdravljenje z NPO samo) in pri katerih zdravljenje z intravensko obliko kemoterapije ni bilo primerno.
Dosage in renally impaired patients
Odmerjanje pri bolnikih z ledvično okvaro
Patients with prior cisplatin chemoradiotherapy Cisplatin
Bolniki s predhodno cisplatinsko kemoradioterapijo topotecan/cisplatin (n = 69)
Myelosuppression leading to sepsis and fatalities due to sepsis have been reported in patients treated with topotecan (see section 4.8).
Pri bolnikih, ki so se zdravili s topotekanom, so poročali o mielosupresiji, ki je povzročila sepso in smrti zaradi sepse (glejte poglavje 4.8).
One Phase 2 study (Study 065) and one Phase 3 study (Study 396) were conducted to evaluate the efficacy of oral topotecan versus intravenous topotecan in patients who had relapsed ≥ 90 days after completion of one prior regimen of chemotherapy (see Table 1).
Za oceno učinkovitosti peroralnega topotekana v primerjavi z intravenskim topotekanom pri bolnikih, ki se jim je pojavil recidiv ≥ 90 dni po dokončanju ene predhodne sheme kemoterapije, je bila opravljena ena študija 2. faze (študija 065) in ena študija 3. faze (študija 396) (glejte preglednico 1).
The clear concentrate is pale yellow in colour and provides 1 mg per ml of topotecan.
Koncentrat je bledo rumen in ima koncentracijo 1 mg topotekana na ml.
oC ± 2
1. dan +
How to use Topotecan Actavis
Kako se uporablja zdravilo Topotekan Actavis
If dose reduction is chosen for patients who experience severe neutropenia (neutrophil count < 0.5 x 10
Če izberete zmanjšanje odmerka za bolnike, ki se jim pojavi huda, sedem ali več dni trajajoča nevtropenija (število nevtrofilcev < 0,5 x 10
severe bleeding (associated with thrombocytopenia) Immune system disorders
Ni znano hude krvavitve (povezane s trombocitopenijo)
Topotecan is not recommended to be used in these patient groups.
Pri teh skupinah bolnikov zdravljenje s topotekanom ni priporočljivo.
Ελλάδα Specifar ABEE
Potem je potreben pregled pri
Fatalities due to neutropenic colitis have been reported in clinical trials with topotecan.
Med kliničnimi preskušanji s topotekanom so poročali o smrtnih izidih zaradi nevtropeničnega kolitisa.
anorexia (which may be severe) Respiratory, thoracic and mediastinal disorders Rare:
Bolezni dihal, prsnega koša in mediastinalnega prostora Redki:
When topotecan (0.75 mg/m2/day for 5 consecutive days) and cisplatin (60 mg/m2/day on Day 1) were administered in 13 patients with ovarian cancer, a slight increase in AUC (12 %, n=9) and Cmax (23 %, n=11) was noted on day 5.
13 bolnicam z rakom jajčnika so 5. dan opazili rahlo povečanje vrednosti AUC (12%, n=9) in Cmax (23%, n=11).
Cisplatin is administered as an intravenous infusion on day 1 at a dose of 50 mg/m2/day and following the topotecan dose.
30 minutne intravenske infuzije enkrat na dan. 1. dan po prejemu odmerka topotekana bolnica prejme še intravensko infuzijo cisplatina v odmerku 50 mg/m2/dan.
Median survival (weeks) (95 % CI) Hazard ratio (95 % CI) Response rate (%) (95 % CI) Difference in response rate (95 % CI) Median time to progression (weeks) (95 % CI) Hazard ratio (95 % CI) N = total number of patients treated.
Mediana vrednost (95 % interval zaupanja) Razmerje tveganja (95 % interval zaupanja)
DATE OF FIRST AUTHORISATION/RENEWAL OF THE AUTHORISATION Date of first authorisation: 24 July 2009
DATUM PRIDOBITVE/PODALJŠANJA DOVOLJENJA ZA PROMET Z
Median survivals for the two groups were 25.0 and 24.7 weeks respectively.
Srednji čas preživetja bolnikov v skupini, ki je prejemala topotekan je bil 25,0 tednov, v skupini, ki je prejemala kombinacijo CAV pa 24,7 tedna.
Oral and intravenous topotecan were associated with similar symptom palliation in patients with relapsed sensitive SCLC in patient selfreports on an unblinded symptom scale assessment in each of these two studies.
Na podlagi neslepljene ocene lestvice simptomov po navedbi bolnikov samih v obeh študijah sta peroralni in intravenski topotekan pri bolnikih z recidivom občutljivega drobnoceličnega pljučnega raka dosegla podobno ublažitev simptomov.
Median (95 % C.I) Hazard ratio (95 % C.I.)
Razmerje tveganja (95 % interval zaupanja) Log rank p-vrednost
Insufficient data are available to make a recommendation for patients with a creatinine clearance <20 ml/min.
Samostojno zdravljenje (drobnocelični pljučni karcinom) Za zdravljenje bolnikov z očistkom kreatinina < 20 ml/min ne moremo priporočiti ustreznih odmerkov, saj imamo premalo izkušenj.
Initial dose The recommended dose of topotecan is 0.75 mg/m2/day administered as 30 minute intravenous infusion daily on days 1, 2 and 3.
Začetni odmerek Priporočeni odmerek topotekana je 0,75 mg/m2/dan.
How to store Topotecan Actavis
Dajanje zdravila Topotekan Actavis
The mean metabolite:parent AUC ratio was less than 10 % for both total topotecan and topotecan lactone.
Tako za skupni topotekan kot topotekan lakton je srednje razmerje AUC-jev presnovek: izvorna učinkovina manjše od 10 %.
In patients (n = 39) with recurrence within 180 days after chemoradiotherapy with cisplatin, the median survival in the topotecan plus cisplatin arm was 4.6 months (95 % C.I.
Pri bolnicah (n = 39) s ponovitvijo v 180 dneh po kemoradioterapiji s cisplatinom je bila mediana vrednost preživetja v skupini, ki je prejemala topotekan in cisplatin, 4,6 meseca (95% interval zaupanja: 2,6; 6,1), v skupini, ki je prejemala cisplatin, pa 4,5 meseca (95% intervala zaupanja: 2,9; 9,6), razmerje tveganja 1,15 (0,59; 2,23).
The prescribing information for cisplatin should be consulted for a full list of adverse events associated cisplatin use.
Za celoten seznam neželenih učinkov, povezanih s cisplatinom, glejte podatke za predpisovanje cisplatina.
ITT population Cisplatin 50 mg/m d.1 q21 d.
Vključene bolnice (ITT populacija) cisplatin 50 mg/m
Topotecan should not be re-administered unless the neutrophil count is ≥ 1 x 10
Nadaljevalni odmerki Pred naslednjo uporabo topotekana mora biti število nevtrofilcev ≥ 1 x 10
malaise
Dojenje
9/l and/or a platelet count of < 100 x 10
9/l in/ali števila
In another randomised phase III trial which compared IV topotecan to cyclophosphamide, doxorubicin and vincristine (CAV) in patients with relapsed, sensitive SCLC, the overall response rate was 24.3 % for topotecan compared to 18.3 % for the CAV group.
Pri bolnikih z relapsom dovzetnega drobnoceličnega pljučnega raka je bil celoten odstotek odziva na topotekan 24,3%, na kombinacijo CAV pa 18,3%.
The elimination of topotecan has only been partly investigated in man. A major route of clearance of topotecan was by hydrolysis of the lactone ring to form the ring-opened carboxylate.
V glavnem se presnavlja s hidrolizo laktonskega obroča do karboksilata z odprtim obročem.
Guidelines for the safe handling and disposal of antineoplastic agents Reconstitution and dilution of the medicinal product must be performed by trained personnel.
Smernice za varno ravnanje in odlaganje antineoplastičnih zdravil
Plasma clearance in patients with renal impairment (creatinine clearance 41-60 ml/min.)
Plazemski očistek pri bolnikih z ledvično okvaro (očistek kreatinina 41−60 ml/min) se je v primerjavi s kontrolno skupino zmanjšal na približno 67%.
The clear concentrate is pale yellow in colour and provides 1 mg per ml of topotecan, as Topotecan Actavis 1 mg contains a 10 % overage of fill.
Ker vsebuje Topotekan Actavis 10% prebitek, ima nastala bledo rumena raztopina koncentracijo 1 mg topotekana na ml.
The adverse event profile for topotecan when given in combination with cisplatin in the cervical cancer clinical trials is consistent with that seen with topotecan monotherapy.
Pri kombiniranem zdravljenju raka materničnega vratu s cisplatinom med kliničnimi preskušanji so bili neželeni učinki topotekana enaki kot pri samostojnem zdravljenju s topotekanom.
STATEMENT OF ACTIVE SUBSTANCE(S) Each vial contains 4 mg topotecan (as hydrochloride).
Ena viala vsebuje 4 mg topotekana (v obliki klorida).
STATEMENT OF ACTIVE SUBSTANCE(S) Each vial contains 1 mg topotecan (as hydrochloride).
Ena viala vsebuje 1 mg topotekana (v obliki klorida).
: 4.9, 9.5) for the cisplatin arm with a hazard ratio of 0.75 (0.49, 1.16).
Pri bolnicah (n = 102) s ponovitvijo po 180 dneh je bila mediana vrednost preživetja v skupini, ki je prejemala topotekan in cisplatin, 9,9 meseca (95% interval zaupanja: 7; 12,6) v skupini, ki je prejemala cisplatin, pa 6,3 meseca (95% interval zaupanja: 4,9; 9,5), razmerje tveganja 0,75 (0,49; 1,16).
Topotecan Actavis is not recommended in case of severe kidney impairment; - if you have liver problems.
Zdravila Topotekan Actavis ni priporočljivo uporabljati pri bolnikih s hudo okvaro ledvic.
NAME OF THE MEDICINAL PRODUCT Topotecan Actavis 4 mg powder for concentrate for solution for infusion
Topotekan Actavis 4 mg prašek za koncentrat za raztopino za infundiranje
NAME OF THE MEDICINAL PRODUCT Topotecan Actavis 4 mg powder for concentrate for solution for infusion topotecan
Topotekan Actavis 4 mg prašek za koncentrat za raztopino za infundiranje topotekan
NAME OF THE MEDICINAL PRODUCT Topotecan Actavis 1 mg powder for concentrate for solution for infusion topotecan
Topotekan Actavis 1 mg prašek za koncentrat za raztopino za infundiranje topotekan
The cellular sequela of inhibition of topoisomerase-I by topotecan is the induction of protein-associated DNA single-strand breaks.
Celična posledica zaviranja topoizomeraze I je razpad posameznih verig DNA, na katere se veže encim, na dele.
CLINICAL PARTICULARS
Pediatrični bolniki
Hazard ratio (95 % C.I.)
Razlika v deležu odziva (95 % IZ)
Not applicable.
če je treba).
NAME OF THE MEDICINAL PRODUCT Topotecan Actavis 1 mg powder for concentrate for solution for infusion
Topotekan Actavis 1 mg prašek za koncentrat za raztopino za infundiranje
There is little or no accumulation of topotecan with repeated daily dosing and there is no evidence of a change in the PK after multiple doses.
Med vsakodnevno uporabo se topotekan kopiči le malo ali sploh ne.
You must tell your doctor immediately if you experience any of the following serious side effects.
Če se vam pojavi kateri od naslednjih resnih neželenih učinkov, morate to nemudoma povedati zdravniku.
anaphylactic reaction angioedema urticaria
prašek za koncentrat za raztopino za infundiranje rumen prašek
Cytotoxic
Topotekan Actavis
Hepatobiliary disorders Common:
Bolezni imunskega sistema Pogosti:
Topotecan Actavis; - if your blood cell counts are too low.
- če imate premajhno število krvnih celic.
What Topotecan Actavis contains - The active substance is topotecan.
Zdravilo Topotekan Actavis vsebuje natrij
This overview was last updated in 12-2018.
Povzetek je bil nazadnje posodobljen 12-2018.
Further information on Orkambi can be found on the Agency's website: ema.europa.eu/medicines/human/EPAR/Orkambi.
Nadaljnje informacije o zdravilu Orkambi so na voljo na spletni strani agencije: ema.europa.eu/medicines/human/EPAR/Orkambi.
The study is to evaluate how the disease progresses in the two groups of children.
Študija naj bi ocenila napredek bolezni pri dveh skupinah otrok.
The tablets or granules are taken every 12 hours with fat-containing foods.
Tablete ali zrnca se jemljejo vsakih 12 ur s hrano, ki vsebuje maščobo.
Orkambi received a marketing authorisation valid throughout the EU on 19 November 2015.
Zdravilo Orkambi je pridobilo dovoljenje za promet, veljavno po vsej Evropski uniji, 19. novembra 2015.
Overall the number of exacerbations was reduced by 39% when compared with placebo.
Celokupno število poslabšanj se je v primerjavi s placebom zmanjšalo za 39 %.
What measures are being taken to ensure the safe and effective use of Orkambi?
Kateri ukrepi se izvajajo za zagotovitev varne in učinkovite uporabe zdravila Orkambi?
Other information about Orkambi
Druge informacije o zdravilu Orkambi
The European Medicines Agency decided that Orkambi's benefits are greater than its risks and recommended that it be approved for use in the EU.
Evropska agencija za zdravila je zaključila, da so koristi zdravila Orkambi večje od z njim povezanih tveganj, in priporočila, da se odobri za uporabo v EU.
The beneficial effects of Orkambi were smaller than expected for a medicine that treats the mechanism of the disease rather than its symptoms.
Koristni učinki zdravila Orkambi so bili manjši, kot bi bilo pričakovati pri zdravilu, ki zdravi mehanizem bolezni, ne njenih simptomov.
As for all medicines, data on the use of Orkambi are continuously monitored.
Tako kot za vsako zdravilo se tudi podatki o uporabi zdravila Orkambi stalno spremljajo.
There was improvement in CFTR protein activity as measured by a decrease in the amount of chloride in sweat after 24 weeks of treatment.
Prišlo je do izboljšanja delovanja beljakovine CFTR, kar je bilo izmerjeno z zmanjšanjem količine klorida v znoju po 24 tednih zdravljenja.
The recommended dose depends on the age and weight of the patient.
Priporočeni odmerek je odvisen od starosti in telesne mase bolnika.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Orkambi have also been included in the summary of product characteristics and the package leaflet.
Priporočila in varnostni ukrepi, ki jih morajo za varno in učinkovito uporabo zdravila Orkambi upoštevati zdravstveni delavci in bolniki, so vključeni tudi v povzetek glavnih značilnosti zdravila in navodilo za njegovo uporabo.
All children were treated with Orkambi.
Vsi otroci so bili zdravljeni z zdravilom Orkambi.
Stopping treatment with Orkambi caused chloride levels to rise again.
S prenehanjem zdravljenja z zdravilom Orkambi je prišlo do ponovnega povečanja ravni klorida.
Orkambi is used in patients who have inherited the mutation from both parents.
Zdravilo Orkambi se uporablja pri bolnikih, ki so podedovali mutacijo od obeh staršev.
However, as cystic fibrosis caused by F508del mutation is particularly severe, the observed effects were considered clinically relevant for patients with no other option.
Ker pa je cistična fibroza, ki jo povzroča mutacija F508del, še posebno težka, je bilo presojeno, da so opaženi učinki za bolnike, ki nimajo druge možnosti, klinično pomembni.
An overview of Orkambi and why it is authorised in the EU
Pregled zdravila Orkambi in zakaj je odobreno v EU
What benefits of Orkambi have been shown in studies?
Kakšne koristi je zdravilo Orkambi izkazalo v študijah?
Treatment with Orkambi also reduced the number of exacerbations (flares-up) requiring hospital admission or antibiotic therapy.
Zdravljenje z zdravilom Orkambi je tudi zmanjšalo število poslabšanj (izbruhov) bolezni, zaradi katerih sta bila potrebna sprejem v bolnišnico ali zdravljenje z antibiotiki.
What are the risks associated with Orkambi?
Kakšna tveganja so povezana z zdravilom Orkambi?
Why is Orkambi authorised in the EU?
Zakaj je zdravilo Orkambi odobreno v EU?
For more information about using Orkambi, see the package leaflet or contact your doctor or pharmacist.
Za več informacij glede uporabe zdravila Orkambi glejte navodilo za uporabo ali se posvetujte z zdravnikom ali farmacevtom.
Side effects reported with Orkambi are carefully evaluated and any necessary action taken to protect patients.
Neželeni učinki, o katerih so poročali pri zdravilu Orkambi, se skrbno ovrednotijo in po potrebi se sprejmejo ukrepi za zaščito bolnikov.
Orkambi's side effects mainly affected the gut and breathing and were generally considered mild to moderate and manageable.
Neželeni učinki zdravila Orkambi so prizadeli predvsem črevesje in dihanje ter so bili večinoma ocenjeni za blage do zmerne in obvladljive.
Serious side effects include liver problems such as raised liver enzymes, cholestatic hepatitis (build-up of bile leading to inflammation of the liver) and hepatic encephalopathy (a brain disease caused by liver problems).
Resni neželeni učinki so vključevali težave z jetri, kot so povišane vrednosti jetrnih encimov, holestatski hepatitis (kopičenje žolča, ki privede do vnetja jeter) in hepatično encefalopatijo (možgansko bolezen, ki jo povzročajo težave z jetri).
The children's growth (measured in terms of the body mass index, weight and height) also improved.
Izboljšala se je tudi rast (izmerjena v obliki indeksa telesne mase, telesne mase in višine).
What is Orkambi and what is it used for?
Kaj je zdravilo Orkambi in za kaj se uporablja?
In total, these serious side effects occurred in more than 1 in 200 people during the clinical studies.
Skupaj so se ti resni neželeni učinki v kliničnih študijah pojavili pri več kot 1 osebi od 200.
The most common side effects with Orkambi (which may affect more than 1 in 10 people) are dyspnoea (shortness of breath), diarrhoea and nausea (feeling sick).
Najpogostejši neželeni učinki zdravila Orkambi (ki lahko prizadenejo več kot 1 osebi od 10) so dispneja (zasoplost), driska in navzeja (siljenje na bruhanje).
Patients with cystic fibrosis have high levels of chloride in sweat due to CFTR not working properly.
Bolniki s cistično fibrozo imajo visoko vsebnost klorida v znoju, ker beljakovina CFTR ne deluje pravilno.
After 24 weeks of treatment, LCI2.5 decreased by 1.01 in patients treated with Orkambi compared with an increase of 0.08 in patients treated with placebo.
Po 24 tednih zdravljenja se je vrednost LCI2.5 pri bolnikih, zdravljenih z zdravilom Orkambi, zmanjšala za 1,01 odstotne točke, v primerjavi z 0,08 pri bolnikih, ki so prejemali placebo.
The mutations reduce the amount of CFTR protein on the cell surface or affect the way the protein works.
Te mutacije zmanjšujejo število beljakovin CFTR na celični površini ali pa vplivajo na način delovanja beljakovine.
It affects the cells that produce mucus and digestive juices.
Prizadene celice, ki proizvajajo sluz in prebavne sokove.
In the study in children aged 6 to 11 years, the main measure of effectiveness was a decrease in the lung clearance index (LCI2.5), which indicates an improvement in lung ventilation.
V študiji pri otrocih, starih od 6 do 11 let, je bilo glavno merilo učinkovitosti znižanje indeksa pljučnega očistka (LCI2.5), ki kaže boljšo predihanost pljuč.
Orkambi can only be prescribed by a doctor with experience in the treatment of cystic fibrosis.
Zdravilo Orkambi smejo predpisovati le zdravniki z izkušnjami z zdravljenjem cistične fibroze.
Orkambi improved lung function in two main studies involving 1,108 cystic fibrosis patients aged 12 years and above, and one study involving 204 children aged from 6 to 11 years.
Zdravilo Orkambi se je izkazalo za učinkovito pri izboljševanju delovanja pljuč v dveh glavnih študijah, ki sta zajeli 1 108 bolnikov s cistično fibrozo, starejših od 12 let, in v eni študiji, ki je zajela 204 otroke, stare od 6 do 11 let.
One of the active substances in Orkambi, lumacaftor, increases the amount of CFTR protein on the cell and the other, ivacaftor, increases the activity of the defective CFTR protein.
Ena od učinkovin v zdravilu Orkambi, lumakaftor, poveča število beljakovin CFTR na celični površini, druga, ivakaftor, pa poveča delovanje okvarjene beljakovine CFTR.
A further long-term study in children aged 2 to 5 years with cystic fibrosis is also required to compare those who have the F508del mutation in the CFTR gene and are treated with Orkambi with those who have not been treated with Orkambi.
Potrebna je tudi nadaljnja dolgoročna študija pri otrocih s cistično fibrozo, starih od 2 do 5 let, da bi se lahko primerjali tisti, ki imajo F508del mutacijo v genu CFTR in se zdravijo z zdravilom Orkambi, s tistimi, ki niso bili zdravljeni s tem zdravilom.
Cystic fibrosis is an inherited disease that has severe effects on the lungs and the digestive system.
Cistična fibroza je dedna bolezen, ki ima resne učinke na pljuča in prebavila.
Orkambi is available as tablets and granules.
Zdravilo Orkambi je na voljo v obliki tablet in zrnc.
For the full list of all side effects and restrictions with Orkambi, see the package leaflet.
Za celoten seznam neželenih učinkov in omejitev pri uporabi zdravila Orkambi glejte navodilo za uporabo.
Orkambi is used in patients who have a genetic mutation (change) called the F508del mutation.
Zdravilo Orkambi se uporablja pri bolnikih z gensko mutacijo (spremembo), imenovano mutacija F508del.
Results from the first study showed that after 24 weeks of treatment patients who took Orkambi had an average improvement in FEV1 of 2.41 percentage points more than those who took placebo; this figure was 2.65 in the second study.
Rezultati prve študije so pokazali, da se je bolnikom, ki so jemali zdravilo Orkambi, po 24 tednih zdravljenja vrednost FEV1 povprečno izboljšala za 2,41 odstotne točke več kot bolnikom, ki so jemali placebo.
The company that markets Orkambi is required to conduct a 5-year study in patients with cystic fibrosis to evaluate the long-term effects of the medicine.
Družba, ki trži zdravilo Orkambi, mora za oceno dolgoročnih učinkov zdravila izvesti petletno študijo pri bolnikih s cistično fibrozo.
In the two studies in patients aged 12 years and above, the main measure of effectiveness was based on improvement in patients' predicted FEV1 which is a measure of how well the lungs work.
Glavno merilo učinkovitosti v obeh študijah pri bolnikih, starejših od 12 let, je temeljilo na izboljšanju odstotka napovedane vrednosti FEV1, kar je merilo, kako dobro pljuča delujejo.
A further study involved 60 children aged 2 to 5 years with cystic fibrosis who had the F508del mutation in the CFTR gene.
V dodatno študijo je bilo vključenih 60 otrok s cistično fibrozo, starih od 2 do 5 let, ki so imeli mutacijo F508del v genu CFTR.
This mutation affects the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR) which is involved in regulating the production of mucus and digestive juices.
Ta mutacija prizadene gen za beljakovino, imenovano regulator transmembranske prevodnosti pri cistični fibrozi (CFTR), ki sodeluje pri uravnavanju nastajanja sluzi in prebavnih sokov.
All patients had the F508del mutation in the CFTR gene.
Vsi bolniki so imeli mutacijo F508del v genu CFTR.
Orkambi is a medicine used to treat cystic fibrosis in patients aged 2 years and above.
Orkambi je zdravilo za zdravljenje cistične fibroze pri bolnikih, starejših od dveh let.
It should be prescribed only to patients confirmed to have the F508del mutation in both copies of the CFTR gene.
Predpisovati se sme le bolnikom, ki imajo potrjeno mutacijo F508del v obeh kopijah gena CFTR.
These actions make mucus and digestive juices less thick.
S tem postanejo sluz in prebavni sokovi manj gosti.
The effects of Orkambi in children aged 2 to 5 years were considered to be similar to those in older children and a long-term study will be carried out to confirm this.
Učinki zdravila Orkambi pri otrocih, starih od 2 do 5 let, so se šteli podobni učinkom pri starejših otrocih.
How is Orkambi used?
Kako se zdravilo Orkambi uporablja?
How does Orkambi work?
Kako zdravilo Orkambi deluje?
This gene makes the CFTR protein, which is involved in regulating the production of mucus and digestive juices.
Ta gen proizvaja beljakovino CFTR, ki sodeluje pri uravnavanju nastajanja sluzi in prebavnih sokov.
Orkambi has been shown to improve lung function and lung ventilation in patients with cystic fibrosis from 6 years of age.
Dokazali so, da zdravilo Orkambi izboljšuje delovanje in predihanost pljuč pri bolnikih s cistično fibrozo, starejših od šest let.
In cystic fibrosis, the secretions become thick and cause blockage.
Pri cistični fibrozi se izločki zgostijo in povzročajo zaporo.
In the studies, Orkambi was compared with placebo (a dummy treatment), both added to patients' usual therapy.
V študijah so zdravilo Orkambi primerjali s placebom (zdravilom brez učinkovine).