FrancophonIA/ELRC3.0 huggingface dataset:
English: It is recommended that the infusion begin at an initial rate of 1 mg/kg/h and be gradually increased by 2 mg/kg/h every 30 minutes if there are no signs of infusion associated reactions (IARs) until a maximum rate of 7 mg/kg/h is reached.
Croatian: Preporučuje se da početna brzina infuzije bude 1 mg/kg/h te da se zatim, ako nema znakova reakcija povezanih s infuzijom, njezina brzina postupno povećava za 2 mg/kg/h svakih 30 minuta dok se ne postigne najveća dopuštena brzina od 7 mg/kg/h.
English: Table 3: Results for endpoint survival using the Cox regression model
Croatian: Tablica 3: Rezultati mjere ishoda preživljenja dobiveni primjenom Coxova regresijskog modela
English: • Inflammation of membrane that covers eyeball and eyelid
Croatian: • upala membrane koja pokriva očnu jabučicu i očni kapak
English: After the first year (64 up to 130 weeks) of treatment LVM further decreased in 8 patients.
Croatian: Nakon prve godine liječenja (64 do 130 tjedana) u 8 se bolesnika masa lijeve klijetke dodatno smanjila.
English: The time course of disease progression is extremely variable and not predictable, with some patients experiencing a rapid deterioration in skeletal and respiratory muscle function leading to loss of ambulation and respiratory failure, others progressing less rapidly, and yet others presenting with a dissociation in the progression of skeletal and respiratory muscle involvement.
Croatian: Vremenski tijek progresije bolesti krajnje je varijabilan i nepredvidljiv, pa tako u nekih bolesnika dolazi do brzog pogoršanja funkcije skeletnih i respiratornih mišića, što dovodi do gubitka sposobnosti kretanja i zatajenja disanja, dok u drugih bolesnika bolest napreduje sporije, a kod nekih čak dolazi do manje zahvaćenosti skeletnih i respiratornih mišića.
English: These proportions were maintained up to the end of the study (with individual patient treatment durations ranging from 1 to 168 weeks; mean follow-up period of 109 weeks).
Croatian: Ovi su se omjeri održali sve do kraja ispitivanja (trajanje liječenja pojedinih bolesnika kretalo se u rasponu od 1 do 168 tjedana, uz prosječno razdoblje praćenja od 109 tjedana).
English: It is noteworthy that a higher proportion of patients with better motor outcomes show stability or improvement in growth parameters (weight), while the large majority of patients, regardless of their motor outcomes or baseline features, show reversal of cardiomyopathy as measured by changes in LVM Z-score.
Croatian: Važno je napomenuti da je u velikog broja bolesnika u kojeg je došlo do poboljšanja motoričkih funkcija zabilježena stabilizacija ili poboljšanje parametara rasta (težina), dok je u velike većine bolesnika, neovisno o poboljšanju motoričkih funkcija ili početnim vrijednostima, došlo do povlačenja kardiomiopatije, što se moglo primijetiti u promjeni Z-vrijednosti mase lijeve klijetke.
English: There are no clinical data on the effects of alglucosidase alfa on fertility.
Croatian: Nema kliničkih podataka o učincima alglukozidaze alfa na plodnost.
English: Patients with Pompe disease treated with immunosuppressive agents maybe at further increased risk of developing severe infections and vigilance is recommended.
Croatian: Bolesnici s Pompeovom bolesti koji se liječe imunosupresivnim lijekovima mogu biti pod dodatnim povećanim rizikom od razvoja teških infekcija te se preporučuje oprez.
English: The remaining 7 patients made no clinically significant motor gains or were unable to sustain the motor gains made and had very limited motor movement by the last study assessment (with individual patient treatment durations ranging from 52 to 142 weeks; mean follow-up period of 103 weeks).
Croatian: Preostalih 7 bolesnika nije ostvarilo klinički značajno motoričko poboljšanje ili nije bilo sposobno održati ostvareno motoričko poboljšanje, pa su na zadnjoj procjeni u sklopu ispitivanja ti bolesnici imali vrlo ograničene motoričke sposobnosti (trajanje liječenja pojedinih bolesnika kretalo se u rasponu od 52 do 142 tjedna, uz prosječno vrijeme praćenja od 103 tjedna).
English: The safety and efficacy of Myozyme in patients with renal or hepatic impairment have not been evaluated and no specific dose regimen can be recommended for these patients.
Croatian: U bolesnika s oštećenjem bubrežne ili jetrene funkcije nisu ocijenjene sigurnost i djelotvornost lijeka Myozyme pa se u tih bolesnika ne može preporučiti poseban režim doziranja.
English: The reconstituted volume is 10.5 ml containing 5 mg/ml, and appears as a clear, colourless to pale yellow solution which may contain particles in the form of thin white strands or translucent fibres.
Croatian: Rekonstituirani volumen od 10,5 ml sadrži 5 mg/ml lijeka i bistra je bezbojna do blijedožuta otopina koja može sadržavati čestice u obliku tankih bijelih niti ili prozirnih vlakana.
English: Patients were randomized in a 2:1 ratio and received 20 mg/kg Myozyme (n=60) or placebo (n=30) once every two weeks for 78 weeks (18 months).
Croatian: 20 mg/kg lijeka Myozyme (n=60) ili placebo (n=30) jedanput svaka dva tjedna tijekom 78 tjedana (18 mjeseci).
English: After a minimum of 52 weeks, 16 of these 18 patients were enrolled in an extension study to receive continued treatment at the same dose for a total duration of up to three years (150 weeks).
Croatian: Nakon najmanje 52 tjedna, 16 od 18 bolesnika bilo je uključeno u produžetak ispitivanja u ukupnom trajanju do 3 godine (150 tjedana) u kojem je liječenje nastavljeno istom dozom.
English: There was no evidence that IgG antibodies to alglucosidase alfa affected pharmacokinetics.
Croatian: Nije bilo dokaza da IgG protutijela na alglukozidazu alfa utječu na farmakokinetiku.
English: Adverse reaction (Preferred Term Level)
Croatian: Nuspojave (razina prema preporučenom izrazu)
English: The recommended dose regimen of alglucosidase alfa is 20 mg/kg of body weight administered once every 2 weeks.
Croatian: Preporučeni režim doziranja alglukozidaze alfa je 20 mg/kg tjelesne težine jedanput svaka 2 tjedna.
English: One vial contains 50 mg of alglucosidase alfa.
Croatian: Jedna bočica sadrži 50 mg alglukozidaze alfa.
English: Intravenous use after reconstitution and dilution
Croatian: Za intravensku primjenu nakon pripreme za primjenu i razrjeđivanja
English: Immunogenicity data from clinical trials and published literature in CRIM-negative infantile-onset patients (IOPD) suggests that the administration of immune tolerance induction (ITI) regimen given to alglucosidase alfa naive patients (prophylactic ITI) may be effective in preventing or reducing the development of High Sustained Antibody Titer (HSAT) against alglucosidase alfa.
Croatian: Podaci o imunogenosti iz kliničkih ispitivanja i objavljene literature u CRIM-negativnih bolesnika s infantilnim oblikom bolesti pokazuju da primjena režima doziranja za indukciju imunološke tolerancije kod bolesnika koji nisu prethodno liječeni alglukozidazom alfa (profilaktička indukcija imunološke tolerancije) može biti učinkovita u prevenciji ili smanjenju razvoja postojanog visokog titra protutijela na alglukozidazu alfa.
English: At 104 weeks of treatment, all 13 patients with available data had maintained or improved weight-for-age percentiles (above the 3rd percentile), all 12 patients with available data were above the 3rd percentile for length and all 12 patients with available data were above the 3rd percentile for head circumference.
Croatian: U 104. tjednu liječenja u svih je 13 bolesnika za koje su podaci bili dostupni zabilježeno poboljšanje ili održavanje vrijednosti percentila težine s obzirom na dob (iznad 3. percentila), visina je iznad 3. percentila bila u svih 12 bolesnika za koje su podaci bili dostupni, a opseg glave bio je iznad 3. percentila u svih 12 bolesnika za koje su podaci bili dostupni.
English: In the untreated historical cohort 5 of 47 patients (10.6%) for whom data were available, were alive at age 30 months (2.5 years).
Croatian: U neliječenoj kohorti bolesnika s prirodnim tijekom bolesti u dobi od 30 mjeseci (2,5 godine) preživjelo je 5 od 47 bolesnika (10,6%) za koje su podaci bili dostupni.
English: Other names for Pompe disease include glycogen storage disease type II (GSDII), acid maltase deficiency (AMD) and glycogenosis type II.
Croatian: Drugi nazivi za Pompeovu bolest su bolest nakupljanja glikogena tipa II, nedostatak kisele maltaze i glikogenoza tipa II.
English: • One study in Germany enrolled 38 late-onset patients with 36 months follow up.
Croatian: • Jedno ispitivanje u Njemačkoj uključivalo je 38 bolesnika s kasnim oblikom Pompeove bolesti, s razdobljem praćenja od 36 mjeseci.
English: Myozyme has to be reconstituted with water for injections, then diluted with sodium chloride 9 mg/ml (0.9%) solution for injection and then administered by intravenous infusion.
Croatian: Myozyme se mora rekonstituirati vodom za injekcije, a zatim razrijediti otopinom natrijeva klorida za injekcije od 9 mg/ml (0,9%) te primijeniti intravenskom infuzijom.
English: Myozyme is given through a drip into a vein (by intravenous infusion).
Croatian: Myozyme se daje infuzijom (dripom) u venu (intravenskom infuzijom).
English: The study data demonstrated that prophylactic ITI prevents or reduces the occurrence of antibodies against alglucosidase alfa over time, which may maintain clinical benefit of ERT and improve survival in CRIM-negative IOPD patients.
Croatian: Podaci iz ispitivanja pokazali su da profilaktička primjena indukcije imunološke tolerancije spriječava ili smanjuje pojavu protutijela na alglukozidazu alfa tijekom vremena, što može održati kliničku korist enzimskog nadomjesnog liječenja i poboljšati preživljenje u CRIM-negativnih bolesnika s infantilnim oblikom Pompeove bolesti.
English: After 52 weeks of treatment 14 of 18 patients (77.8%) had maintained or improved weight-for-age percentiles (above the 3rd percentile), 14 of 15 patients (93.3%) were above the 3rd percentile for length and 12 of 15 patients (80.0%) were above the 3rd percentile for head circumference.
Croatian: Nakon 52 tjedna liječenja, percentil vrijednosti težine s obzirom na dob (iznad 3. percentila) održao se ili poboljšao u 14 od 18 bolesnika (77,8%), visina je bila iznad 3. percentila u 14 od 15 bolesnika (93,3%), a opseg glave iznad 3. percentila u 12 od 15 bolesnika (80,0%).
English: There is no experience with overdose of alglucosidase alfa.
Croatian: Nema iskustva s predoziranjem alglukozidazom alfa.
English: Reactions in 2 of these patients were IgE-mediated hypersensitivity reactions.
Croatian: Reakcije u 2 navedena bolesnika bile su reakcije preosjetljivosti u kojima je posredovao IgE.
English: Patients with an acute illness (e.g. pneumonia, sepsis) at the time of Myozyme infusion appear to be at greater risk for IARs.
Croatian: U bolesnika s akutnom bolešću u vrijeme primanja infuzije lijeka Myozyme (npr. pneumonijom, sepsom) postoji veći rizik od pojave reakcija povezanih s infuzijom.
English: Life threatening reactions, including very severe generalised allergic reactions and anaphylactic shock, have been reported in some patients.
Croatian: U nekih bolesnika prijavljene su po život opasne reakcije, uključujući vrlo teške generalizirane alergijske reakcije i anafilaktički šok.
English: Therefore, a 0.2 micron low protein binding in-line filter should be used for administration.
Croatian: Stoga za primjenu infuzije treba upotrijebiti “in-line” filtar od 0,2 µm male sposobnosti vezanja proteina.
English: Patients with infantile-onset Pompe disease who demonstrated motor gains, had greater preservation of motor function and lower glycogen content in the quadriceps muscle at baseline.
Croatian: Bolesnici s infantilnim oblikom Pompeove bolesti u kojih je došlo do motoričkih poboljšanja imali su bolje očuvane motoričke funkcije i nižu početnu vrijednost glikogena u mišiću kvadricepsa.
English: Instructions for use - reconstitution, dilution and administration
Croatian: Sljedeće informacije namijenjene su samo zdravstvenim radnicima: Upute za uporabu - rekonstitucija, razrjeđivanje i primjena
English: Additional efficacy data showed that of 16 patients who were free of invasive-ventilator support at baseline, 7 remained so after 104 weeks of treatment.
Croatian: Dodatni podaci o djelotvornosti pokazali su da u 7 od 16 bolesnika koji na početku liječenja nisu trebali invazivnu mehaničku ventilaciju potrebe za ventilacijom nije bilo ni nakon 104 tjedna liječenja.
English: The primary endpoint was the proportion of patients who were alive and free of invasive ventilator support.
Croatian: Primarna mjera ishoda bila je udio preživjelih bolesnika kojima nije bila potrebna invazivna mehanička ventilacija.
English: After 78 weeks, patients treated with Myozyme showed improvement in distance walked as measured by 6MWT and stabilization of pulmonary function as measured by FVC % predicted as compared to placebo-treated patients.
Croatian: Nakon 78 tjedana su, u usporedbi s bolesnicima koji su primali placebo, u bolesnika koji su primali Myozyme primijećeni napredak u prijeđenoj udaljenosti mjerenoj testom 6MWT i stabilizacija plućne funkcije mjerena postotkom predviđenog FVC-a.
English: Symptoms of life-threatening allergic reactions include, but are not limited to, low blood pressure, very fast heart rate, difficulty breathing, vomiting, facial swelling, hives or rash.
Croatian: Simptomi po život opasne alergijske reakcije uključuju, ali nisu ograničeni na, niski krvni tlak, vrlo brzi srčani otkucaji, otežano disanje, povraćanje, oticanje lica, koprivnjaču ili osip.
English: Some patients have been successfully rechallenged and continued to receive alglucosidase alfa under close clinical supervision.
Croatian: U nekih je bolesnika uspješno uvedeno ponovno liječenje pa su nastavili primati alglukozidazu alfa pod pomnim kliničkim nadzorom.
English: -The active substance is alglucosidase alfa.
Croatian: - Djelatna tvar je alglukozidaza alfa.
English: *One patient who did not have data post baseline was excluded from the analyses.
Croatian: * Iz analize je isključen jedan bolesnik za kojeg nisu bili dostupni podaci o vrijednostima nakon početka liječenja.
English: The probability of a poor outcome and of developing high and sustained IgG antibody titres appears higher among CRIM-negative patients (Cross Reactive Immunologic Material- negative patients in whom no endogenous GAA protein was detected by Western blot analysis) than among CRIM-positive patients in whom endogenous GAA protein was detected by Western blot analysis and/or predicted based on genotype.
Croatian: Cross Reactive Immunologic Material - negativni bolesnici; u kojih Western blot analizom nije otkriven endogeni GAA protein) nego u CRIM-pozitivnih bolesnika (u kojih je endogeni GAA protein otkriven Western blot analizom i/ili predviđen na osnovi genotipa).
English: Infusions should be administered incrementally.
Croatian: Brzinu infuzije treba postupno povećavati.
English: 3 Reactions reported more frequently in the placebo group than in the Myozyme group in late-onset patients.
Croatian: 3 Reakcije prijavljene češće u skupini koja je primala placebo nego u skupini koja je liječena lijekom Myozyme u bolesnika s kasnim oblikom bolesti.
English: The remaining 5 patients showed no significant change in motor development beyond Week 52 (with individual patient treatment durations ranging from 104 to 168 weeks; mean follow-up period of 140 weeks), including 4 patients with no significant motor skills in any of the positions evaluated and 1 patient with only functional sitting skills by the last study visit.
Croatian: U preostalih 5 bolesnika nisu utvrđene značajne promjene u motoričkom razvoju nakon 52 tjedna liječenja (trajanje liječenja pojedinih bolesnika kretalo se u rasponu od 104 do 168 tjedana, uz prosječno razdoblje praćenja od 140 tjedana), uključujući 4 bolesnika koji na posljednjem pregledu u sklopu ispitivanja nisu imala značajne motoričke sposobnosti ni u jednom od vrednovanih položaja i 1 bolesnika koji je imao samo funkcionalnu sposobnost sjedenja.
English: A small number of patients (<1%) in clinical trials and in the commercial setting developed anaphylactic shock and/or cardiac arrest during Myozyme infusion that required life-support measures.
Croatian: U kliničkim ispitivanjima i nakon stavljanja lijeka u promet u malog su se broja bolesnika (< 1%) tijekom infuzije lijeka Myozyme javili anafilaktički šok i/ili zastoj srca koji su zahtijevali mjere oživljavanja.
English: Should you experience any reaction like this, please tell your doctor immediately.
Croatian: Ako razvijete takvu reakciju, o tome odmah obavijestite svog liječnika.
English: An additional 4 patients made motor development gains during the study and were sitting independently by the last study assessment (with individual patient treatment durations ranging from 78 to 130 weeks; mean follow-up period of 110 weeks), although they did not have functional use of the legs.
Croatian: Dodatna 4 bolesnika ostvarila su motorički napredak tijekom ispitivanja te su u posljednjoj procjeni u sklopu ispitivanja mogla samostalno sjediti (trajanje liječenja pojedinih bolesnika kretalo se u rasponu od 78 do 130 tjedana, uz prosječno vrijeme praćenja od 110 tjedana), iako nisu mogla funkcionalno upotrebljavati noge.
English: IARs are defined as any related adverse event occurring during the infusion or during the hours following infusion.
Croatian: Reakcijom povezanom s infuzijom smatra se svaki povezani štetni događaj koji se javi tijekom infuzije ili u satima nakon nje.
English: The infantile-onset form of Pompe disease is characterised by massive deposition of glycogen in the heart, and skeletal muscle always resulting in rapidly progressive cardiomyopathy, generalised muscle weakness and hypotonia.
Croatian: Značajka infantilnog oblika Pompeove bolesti je nakupljanje velike količine glikogena u srcu i skeletnim mišićima, što uvijek dovodi do brzo napredujuće kardiomiopatije, generalizirane slabosti mišića i hipotonije.
English: Better treatment responses were observed in younger patients with less advanced disease who received prophylactic ITI before development of HSAT, which suggests that early initiation of ITI can result in improved clinical outcomes.
Croatian: Bolji odgovori na postupak zapaženi su kod mlađih bolesnika s manje uznapredovalom bolesti u kojih je provedena profilaktička indukcija imunološke tolerancije prije nego što su razvili postojani visoki titar protutijela, što ukazuje da rano provođenje indukcije imunološke tolerancije može rezultirati poboljšanim kliničkim ishodima.
English: A. MANUFACTURERS OF THE BIOLOGICAL ACTIVE SUBSTANCE AND MANUFACTURERS RESPONSIBLE FOR BATCH RELEASE
Croatian: PROIZVOĐAČI BIOLOŠKE DJELATNE TVARI I PROIZVOĐAČI ODGOVORNI ZA
English: Patients who have experienced IARs (and in particular anaphylactic reactions) should be treated with caution when re-administering Myozyme (see sections 4.3 and 4.8).
Croatian: U bolesnika u kojih su se javile reakcije povezane s infuzijom (posebno anafilaktičke reakcije) potreban je poseban oprez kod ponovne primjene lijeka Myozyme (vidjeti dijelove 4.3 i 4.8).
English: You are recommended to stop breast-feeding when you are given Myozyme.
Croatian: Preporučuje se prekinuti dojenje tijekom primjene lijeka Myozyme.
English: Eye disorders not known
Croatian: Poremećaji oka nepoznato konjunktivitis
English: An open-label clinical trial assessed the safety and efficacy of Myozyme in 5 patients with late-onset Pompe disease who ranged in age from 5 to 15 years at initiation of treatment (AGLU02804).
Croatian: Sigurnost i djelotvornost lijeka Myozyme procijenjene su u otvorenom kliničkom ispitivanju u kojem je sudjelovalo 5 bolesnika s kasnim oblikom Pompeove bolesti koji su u trenutku početka liječenja bili u dobi od 5 do 15 godina (AGLU02804).
English: Pompe disease
Croatian: Pompeova bolest
English: • One study in the Netherlands enrolled 69 late-onset patients with a median follow-up of 23
Croatian: • Jedno ispitivanje u Nizozemskoj uključivalo je 69 bolesnika s kasnim oblikom Pompeove bolesti, s medijanom praćenja od 23 mjeseca.
English: How to store Myozyme
Croatian: Kako čuvati Myozyme
English: There are no data from the use of alglucosidase alfa in pregnant women.
Croatian: Nema podataka o primjeni alglukozidaze alfa u trudnica.
English: • Irritability
Croatian: • razdražljivost
English: At 104 weeks of treatment LVM assessments were available for 10 patients, of which 9 decreased to within normal limits.
Croatian: U 104. tjednu liječenja masu lijeve klijetke bilo je moguće odrediti u 10 bolesnika, od kojih su u njih 9 vrijednosti bile u granicama normale.
English: • Decreased heart rate
Croatian: • smanjena brzina srčanih otkucaja
English: Comparison of survival curves from time of diagnosis versus the untreated historical cohort was made using a Cox proportional hazards regression analysis.
Croatian: Krivulje preživljenja od trenutka postavljanja dijagnoze u odnosu na neliječenu kohortu bolesnika s prirodnim tijekom bolesti uspoređene su primjenom Coxove regresijske analize razmjernih rizika.
English: Adverse reactions were mostly mild to moderate in intensity and almost all occurred during the infusion or during the 2 hours following the infusion (infusion associated reactions, IARs).
Croatian: Nuspojave su uglavnom bile blagog do umjerenog intenziteta i gotovo su sve nastupile tijekom infuzije ili unutar 2 sata nakon primjene infuzije
English: Of the 21 patients, 16 survived through the end of this study, with a median time from ERT initiation to last assessment of 44.6 months (range: 5.7 to 105.47); 5 patients died due to respiratory failure and disease progression, all of whom were ERTnaïve at the start of ERT+ITI treatment.
Croatian: Od 21 bolesnika, njih 16 je preživjelo do kraja ovog ispitivanja, s medijanom vremena od započinjanja enzimskog nadomjesnog liječenja do zadnje procjene od 44,6 mjeseci (raspon: 5,7 do 105,47); 5 bolesnika je umrlo zbog zatajenja dišnog sustava i napredovanja bolesti, a od kojih niti jedan nije prethodno primao enzimsko nadomjesno liječenje u trenutku kada je započeto liječenje enzimskim nadomjesnim liječenjem i indukcija imunološke tolerancije.
English: Patients who develop IgE antibodies to alglucosidase alfa appear to be at a higher risk for the occurrence of IARs when Myozyme is readministered (see section 4.8).
Croatian: U bolesnika koji razviju IgE protutijela na alglukozidazu alfa postoji veći rizik od pojave reakcija povezanih s infuzijom pri ponovnoj primjeni lijeka Myozyme (vidjeti dio 4.8).
English: There was no difference in the pharmacokinetic profile of alglucosidase alfa in these juvenile lateonset patients compared to infantile-onset patients.
Croatian: U ovih mladih bolesnika s kasnim oblikom bolesti nije bilo razlike u farmakokinetičkom profilu alglukozidaze alfa u usporedbi s bolesnicima s infantilnim oblikom bolesti.
English: Nephrotic syndrome as well as severe cutaneous reactions, possibly immune mediated, have been reported with alglucosidase alfa including ulcerative and necrotizing skin lesions (see section 4.4).
Croatian: Kod primjene alglukozidaze alfa prijavljeni su nefrotski sindrom i teške kožne reakcije, možda imunološki posredovane, uključujući ulcerativne i nekrotizirajuće kožne lezije (vidjeti dio 4.4).
English: Myozyme is a powder for concentrate for solution for infusion in a vial (50 mg/vial).
Croatian: Myozyme je prašak za koncentrat za otopinu za infuziju u bočici (50 mg/bočica).
English: Patients presented with a constellation of signs and symptoms, primarily respiratory, cardiovascular, edematous and/or cutaneous in nature (see section 4.4).
Croatian: prvenstveno respiratornih, kardiovaskularnih, edematoznih i/ili kožnih (vidjeti dio 4.4).
English: • Eyes tearing
Croatian: • suzenje očiju