natgillin/translations-raw huggingface dataset:
If severe allergic reactions occur, your doctor will stop the infusion immediately, and start giving you suitable treatment.
Če se pojavijo hude alergijske reakcije, bo zdravnik prekinil infundiranje in takoj začel ustrezno zdravljenje.
Clinical study HGT-ELA-038 evaluated immunogenicity in children 16 months to 7.5 years of age.
V klinični študiji HGT-ELA-038 so ocenili imunogenost pri otrocih, starih od 16 mesecev do 7,5 leta.
Four patients experienced a hypoxic episode during one or several infusions, which necessitated oxygen therapy in 3 patients with severe underlying obstructive airway disease (2 with a pre-existing tracheostomy).
Pri štirih bolnikih je med enim ali več infundiranji prišlo do hipoksične epizode, zaradi katerih je bilo zdravljenje s kisikom nujno pri treh bolnikih z obstoječo obstruktivno boleznijo dihalnih poti (dva s predhodno traheotomijo).
After 105 weeks of exposure to idursulfase, neither antibody status nor genotype affected reductions in liver and spleen size or distance walked in the 6-minute walk test or forced vital capacity measurements.
Po 105 tednih izpostavljenosti idursulfazi stanje protiteles in genotip nista vplivala na zmanjšanje velikosti jeter ter vranice, razdaljo, prehojeno pri 6-minutnem testu hoje, in forsirane meritve vitalne kapacitete.
This causes cells to function abnormally, thereby causing problems for various organs in your body which can lead to tissue destruction and organ malfunction and failure.
To povzroči nenormalno delovanje celic, kar povzroča težave v različnih organih v telesu, to pa lahko povzroči uničenje tkiv ter nepravilno delovanje in odpoved organov.
Caution must be exercised in the management and treatment of such patients by limitation or careful monitoring of antihistamine and other sedative medicinal product use.
Pri zdravljenju takšnih bolnikov je treba biti previden in omejiti ali skrbno nadzorovati uporabo antihistaminikov in drugih sedativov.
If you have experienced severe or potentially life-threatening allergic-type reactions to idursulfase or any of the other ingredients of this medicine (listed in section 6) and these cannot be controlled with appropriate medical treatment.
- če ste doživeli hude ali potencialno smrtno nevarne alergijske reakcije na idursulfazo ali katero koli sestavino tega zdravila (navedeno v poglavju 6) in jih ni mogoče nadzorovati z ustreznim zdravljenjem.
Specific Obligation 1 - Hunter Outcome Survey (HOS): available data and updates will be provided within Annual Reassessments.
Posebna obveznost 1 - Raziskava rezultatov pri Hunterjevem sindromu (HOSHunter Outcome Survey): dostopni podatki in posodobitve se bodo predložili z vsakoletnimi ponovnimi ocenami.
Results from pivotal clinical study at 0.5 mg/kg per week (Study TKT024).
Rezultati iz ključne klinične študije ob uporabi odmerka 0,5 mg/kg na teden (študija TKT024).
The overall neutralizing antibody rate was 26/107 patients (24%).
Nevtralizirajoča protitelesa so se razvila pri skupno 26/107 bolnikov (24 %).
It is not known whether idursulfase is excreted in human breast milk.
Ni znano, ali se idursulfaza izloča v materino mleko.
▼This medicine is subject to additional monitoring.
▼Za to zdravilo se izvaja dodatno spremljanje varnosti.
% Change in spleen volume
% spremembe prostornine vranice
Do not use this medicine after the expiry date which is stated on the label and carton after EXP.
Tega zdravila ne smete uporabljati po datumu izteka roka uporabnosti, ki je naveden na nalepki in škatli poleg oznake »Uporabno do«.
If you forget to use Elaprase
Če ste pozabili uporabiti zdravilo Elaprase
As a precautionary measure, it is preferable to avoid the use of idursulfase during pregnancy.
Iz previdnostnih razlogov se je med nosečnostjo uporabi idursulfaze bolje izogibati.
The higher the urinary GAG levels at baseline, the greater the magnitude of decreases in urinary GAG with idursulfase treatment.
Višje kot so ravni GAG v urinu na izhodišču, večje je zmanjšanje ravni GAG v urinu ob zdravljenju z idursulfazo.
The frequency of infusion-related reactions decreased over time with continued treatment.
Pogostnost učinkov, povezanih z infundiranjem, se je ob nadaljevanju zdravljenja sčasoma manjšala.
In some patients glycosaminoglycan accumulates also in the brain.
Pri nekaterih bolnikih se glikozaminoglikan kopiči tudi v možganih.
The longer term effects of antibody development on response to treatment have not been established.
Dolgoročni učinki razvoja protiteles na odziv na zdravljenje še niso ugotovljeni.
Most side effects are mild to moderate and associated with the infusion, however some side effects may be serious.
Večina neželenih učinkov je blagih do zmernih in povezanih z infundiranjem, nekateri neželeni učinki so lahko resni.
A decision must be made whether to discontinue breast-feeding or to discontinue/abstain from idursulfase therapy taking into account the benefit of breast feeding for the child and the benefit of therapy for the woman.
Odločiti se je treba med prenehanjem dojenja in prenehanjem/prekinitvijo zdravljenja z idursulfazo, pri čemer je treba pretehtati prednosti dojenja za otroka in prednosti zdravljenja za mater.
Across clinical studies, serious adverse reactions were reported in a total of 5 patients who received 0.5 mg/kg weekly or every other week.
V vseh kliničnih študijah so o resnih neželenih učinkih poročali pri skupaj petih bolnikih, ki so prejemali 0,5 mg/kg na teden ali na dva tedna.
FVC absolute volume (L)
Absolutna prostornina FVK (l)
Since more than one infusion-related reaction may have occurred during any single infusion, the above numbers are likely to overestimate the true incidence of infusion reactions.
Med enim infundiranjem se lahko pojavi več kot en učinek, povezan z infundiranjem, zato so navedene številke verjetno večje od dejanske incidence učinkov pri infundiranju.
Calculate the total dose to be administered and number of Elaprase vials needed.
Izračunajte skupni odmerek, ki ga boste uporabili, in število potrebnih vial zdravila Elaprase.
Additional clinical benefit analyses were performed on individual components of the primary endpoint composite score, absolute changes in FVC, changes in urine GAG levels, liver and spleen volumes, measurement of forced expiratory volume in 1 second (FEV1), and changes in left ventricular mass (LVM).
Dodatne analize klinične koristi so opravili na posameznih komponentah sestavljenega rezultata primarnega cilja študije, absolutnih spremembah FVK, spremembah ravni GAG v urinu, prostornini jeter in vranice, meritvah forsiranega ekspiracijskega volumna v prvi sekundi (FEV1) in spremembi mase levega ventrikla (LVM).
What Elaprase is and what it is used for
Kaj je zdravilo Elaprase in za kaj ga uporabljamo
Patients with the most severe pulmonary impairment at baseline (as measured by % predicted FVC) tended to show the least improvement.
Pri bolnikih z najhujšo pljučno odpovedjo na izhodišču (merjeno z odstotkom predvidene FVK) se je pokazalo najmanjše izboljšanje.
In addition, mean liver volume decreased to a near maximum extent by Month 8 in all patients previously treated, with a slight increase observed at Month 36.
Poleg tega se je srednja prostornina jeter zmanjšala na skoraj najmanjšo možno do osmega meseca pri vseh bolnikih, ki so jih predhodno zdravili, pri čemer so v 36. mesecu opazili rahlo povečanje.
The study included patients with a documented deficiency in iduronate-2sulfatase enzyme activity, a percent predicted FVC <80%, and a broad spectrum of disease severity.
V študiji so sodelovali bolniki z dokumentiranim pomanjkanjem dejavnosti encima iduronat-2-sulfataze, z odstotkom pričakovane vrednosti FVK < 80 % in širokim spektrom stopenj bolezni.
Available data in animals have shown excretion of idursulfase in milk (see section 5.3).
Razpoložljivi podatki pri živalih kažejo na izločanje idursulfaze v mleko (glejte poglavje 5.3).
These patients should be closely monitored and infused in an appropriate clinical setting.
Te bolnike je treba pozorno spremljati, infundiranje pa mora biti opravljeno v ustrezno opremljenih prostorih.
Idursulfase has no or negligible influence on the ability to drive and use machines.
Idursulfaza nima vpliva ali ima zanemarljiv vpliv na sposobnost vožnje in upravljanja s stroji.
This medicine is a concentrate for solution for infusion.
To zdravilo je koncentrat za raztopino za infundiranje.
Adverse reactions are listed in table 1 with information presented by system organ class and frequency.
Neželeni učinki na zdravilo so navedeni v tabeli 1, podatki pa so prikazani po organskih sistemih in pogostnosti.
The decreases in liver and spleen volumes observed at the end of study TKT024 (week 53) were maintained during the extension study (TKT024EXT) in all patients regardless of the prior treatment they had been assigned.
Zmanjšanje prostornine jeter in vranice, opaženo na koncu študije TKT024 (53. teden), se je vzdrževalo med podaljšano študijo (TKT024EXT) pri vseh bolnikih, ne glede na predhodno obliko zdravljenja, ki jim je bila dodeljena.
Severe or life-threatening hypersensitivity to the active substance or to any of the excipients listed in section 6.1 if hypersensitivity is not controllable.
Huda ali smrtno nevarna preobčutljivost na učinkovino ali katero koli pomožno snov, navedeno v poglavju 6.1, če preobčutljivosti ni mogoče nadzorovati.
Patients in the TKT024 and HGT-ELA-038 studies were also stratified across five weight categories; as shown in the following table:
Bolnike v študijah TKT024 in HGT-ELA-038 so razvrstili tudi v pet kategorij telesne mase, kot je prikazano v spodnji preglednici:
Uncommon side effects (may affect up to 1 in 100 people) are: • Quickened breathing
Občasni neželeni učinki (pojavijo se pri največ 1 od 100 bolnikov) so: • pospešeno dihanje
Elaprase is used as enzyme replacement therapy to treat children and adults with Hunter syndrome (Mucopolysaccharidosis II) when the level of the enzyme iduronate-2-sulfatase in the body is lower than normal, helping improve the symptoms of the disease.
Zdravilo Elaprase se uporablja kot encimsko nadomestno zdravljenje za zdravljenje otrok in odraslih s Hunterjevim sindromom (mukopolisharidozo II), kadar je količina encima iduronat-2-sulfataze v telesu manjša od normalne, za izboljšanje simptomov bolezni.
- The calculated volume of Elaprase should be withdrawn from the appropriate number of vials.
- Vzemite izračunani volumen zdravila Elaprase iz ustreznega števila vial.
The longer-term effects of antibody development on clinical outcomes have not been established.
Dolgotrajnih učinkov razvoja protiteles na klinične izide niso ugotovili.
This medicine has no or negligible influence on the ability to drive and use machines.
To zdravilo nima vpliva ali ima zanemarljiv vpliv na sposobnost upravljanja vozil in strojev.
What you need to know before you use Elaprase
Kaj morate vedeti, preden boste uporabili zdravilo Elaprase
The most severe episode occurred in a patient with a febrile respiratory illness and was associated with hypoxia during the infusion, resulting in a short seizure.
Najhujša epizoda se je pojavila pri bolniku s febrilno boleznijo dihal, povezana pa je bila s hipoksijo med infundiranjem, ki je povzročila kratek epileptični napad.
Other medicines and Elaprase
Druga zdravila in zdravilo Elaprase
In the fourth patient, who had less severe underlying disease, spontaneous resolution occurred shortly after the infusion was interrupted.
Četrti bolnik, ki je imel manj hudo obstoječo bolezen, je kmalu po prekinitvi infundiranja spontano okreval.
Liver volume normalised by Month 24 for 73% (52 out of 71) of patients with hepatomegaly at baseline.
Prostornina jeter se je do 24. meseca normalizirala pri 73 % (52 od 71) bolnikov s hepatomegalijo na izhodišču.
Idursulfase has a molecular weight of approximately 76 kD.
Molska masa idursulfaze je približno 76 kD.
Spleen volume normalised by Months 12 and 24 for 9.7% of patients in the TKT024 Weekly group with splenomegaly.
Prostornina vranice se je do 12. in 24. meseca normalizirala pri 9,7 % bolnikov s splenomegalijo v skupini TKT024 enkrat na teden.
Antibodynegative patients were found exclusively in the missense mutation genotype group (see sections 4.4 and 5.1).
Negativne izvide testov za protitelesa so dobili izključno v skupini bolnikov z genotipom z »missense« (drugačnosmiselno) mutacijo (glejte poglavji 4.4 in 5.1).
The safety and efficacy of Elaprase has been shown in three clinical studies: two randomised, placebo-controlled clinical studies (TKT008 and TKT024) in adults and children above the age of 5 years and one open-label, safety study (HGT-ELA-038) in children between the age of 16 months and 7.5 years.
Varnost in učinkovitost zdravila Elaprase so dokazali v treh kliničnih študijah: dveh randomiziranih, s placebom nadzorovanih kliničnih študijah (TKT008 in TKT024) pri odraslih in otrocih, starejših od 5 let, in eni odprti študiji varnosti (HGT-ELA-038) pri otrocih, starih od 16 mesecev do 7,5 leta.
Excipient with known effect Each vial contains 0.482 mmol of sodium.
Pomožna snov z znanim učinkom Ena viala vsebuje 0,482 mmol natrija.
This medicine will be given to you under the supervision of a doctor or nurse who is knowledgeable in the treatment of Hunter syndrome or other inherited metabolic disorders.
To zdravilo boste prejeli pod nadzorom zdravnika ali medicinske sestre z izkušnjami pri zdravljenju Hunterjevega sindroma ali drugih dednih presnovnih bolezni.
The primary efficacy endpoint was a two-component composite score based on the sum of the ranks of the change from baseline to the end of the study in the distance walked during six minutes (6minute walk test or 6MWT) as a measure of endurance, and % predicted forced vital capacity (FVC) as a measure of pulmonary function.
Primarni cilj študije je bil dvokomponentni rezultat, ki je temeljil na vsoti ranga spremembe razdalje, prehojene v 6 minutah (6-minutni preizkus s hojo ali 6MWT - „6 minute walk test“) kot mere za vzdržljivost in odstotka pričakovane forsirane vitalne kapacitete (FVK) kot mere za delovanje pljuč.
Among all patients, mean % predicted FVC was significantly increased at Month 16, although by Month 36, it was similar to the baseline.
Pri vseh bolnikih je bil srednji odstotek predvidene FVK pomembno povečan v 16. mesecu, čeprav je bil do 36. meseca podoben kot na izhodišču.
Method of administration For instructions on dilution of the medicinal product before administration, see section 6.6.
Način uporabe Za navodila glede redčenja zdravila pred dajanjem glejte poglavje 6.6.
Due to the missing or defective iduronate-2-sulfatase enzyme in patients with Hunter syndrome, glycosaminoglycans progressively accumulate in the cells, leading to cellular engorgement, organomegaly, tissue destruction, and organ system dysfunction.
Zaradi popolnega ali delnega pomanjkanja encima iduronat-2-sulfataze pri bolnikih s Hunterjevim sindromom se glikozaminoglikani progresivno kopičijo v celicah, kar povzroča povečanje celic, organomegalijo, razkroj tkiv in okvaro delovanja organskih sistemov.
For instructions for use, see section 6.6.
Za navodila za uporabo glejte poglavje 6.6.
Special care should be taken when administering an infusion in patients with severe underlying airway disease.
Posebno je treba biti pozoren pri infundiranju pri bolnikih, ki imajo hudo bolezen dihalnih poti.
Please consult your doctor for additional information.
Dodatne informacije lahko dobite pri zdravniku.
Of these 6 (40%) had normalised LVM by the end of the study.
Pri šestih bolnikih med njimi (40 %) se je do konca študije vrednost LVM povrnila na normalno vrednost.
If you suffer from Hunter syndrome, a carbohydrate called glycosaminoglycan which is normally broken down by your body, is not broken down and slowly accumulates in various organs in your body.
Če imate Hunterjev sindrom, se ogljikov hidrat, imenovan glikozaminoglikan, ki se sicer razgrajuje v telesu, ne razgrajuje in se počasi kopiči v različnih celicah v telesu.
This treatment should be supervised by a physician or other healthcare professional experienced in the management of patients with MPS II disease or other inherited metabolic disorders.
Zdravljenje mora nadzorovati zdravnik ali drug zdravstveni delavec z izkušnjami pri obravnavanju bolnikov z MPS II ali drugimi dednimi presnovnimi boleznimi.
The decreases in mean liver volume were seen regardless of age, disease severity, IgG antibody status or neutralising antibody status.
Zmanjšanje srednje prostornine jeter se je pojavilo ne glede na starost, resnost bolezni, stanje protiteles IgG ali stanje nevtralizirajočih protiteles.
Severe or potentially life-threatening hypersensitivity is a contraindication to rechallenge, if hypersensitivity is not controllable (see section 4.3).
Huda ali potencialno smrtno nevarna preobčutljivost je kontraindikacija za ponovno zdravljenje, če preobčutljivosti ni mogoče nadzorovati (glejte poglavje 4.3).
• Whenever the risk management system is modified, especially as the result of new information being received that may lead to a significant change to the benefit/risk profile or as the result of an important (pharmacovigilance or risk minimisation) milestone being reached.
Posodobljen RMP je treba predložiti: ▪ na zahtevo Evropske agencije za zdravila; ▪ ob vsakršni spremembi sistema za obvladovanje tveganj, zlasti kadar je tovrstna sprememba posledica prejema novih informacij, ki lahko privedejo do znatne spremembe razmerja med koristmi in tveganji, ali kadar je ta sprememba posledica tega, da je bil dosežen pomemben mejnik (farmakovigilančni ali povezan z zmanjševanjem tveganja).
• Pain in the joints
• bolečina v sklepih
Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow specific binding of the enzyme to the M6P receptors on the cell surface, leading to cellular internalization of the enzyme, targeting to intracellular lysosomes and subsequent catabolism of accumulated GAG.
Manoza-6-fosfatne (M6P) molekule na oligosaharidnih verigah omogočajo specifično vezavo encima na receptorje za M6P na celični površini, zaradi česar lahko encim preide v celico, kjer prispe do lizosomov in omogoči katabolizem nakopičenih GAG.
There is no experience with overdoses of this medicine.
Izkušenj s prevelikim odmerjanjem tega zdravila ni.
Elaprase is supplied in pack sizes of 1, 4 and 10 vials per carton.
Zdravilo Elaprase je na voljo v velikostih pakiranja po 1, 4 in 10 vial na škatlo.
No clinical data exist demonstrating a benefit on the neurological manifestations of the disorder.
Kliničnih podatkov o pozitivnem vplivu na nevrološke znake bolezni ni.
Do not use Elaprase
Ne uporabljajte zdravila Elaprase
Home infusions should be performed under the surveillance of a physician or other healthcare professional.
Infundiranje na domu mora nadzorovati zdravnik ali drug zdravstveni delavec.
The patients in the weekly treatment group experienced a clinically significant 15% mean improvement in FEV1 at the end of the study.
Pri bolnikih, ki so zdravljenje prejemali enkrat tedensko, je bilo klinično pomembno izboljšanje srednje vrednosti FEV1 na koncu študije 15-odstotno.
• Abdominal pain, nausea, vomiting, frequent and/or loose stools
• bolečine v trebuhu, slabost, bruhanje, pogosto in/ali redko blato
Across 4 clinical studies (TKT008, TKT018, TKT024 and TKT024EXT), 53/107 patients (50%) developed anti-idursulfase IgG antibodies at some point.
V štirih študijah (TKT008, TKT018, TKT024 in TKT024EXT) so se pri 53/107 bolnikov (50 %) razvila protitelesa IgG proti idursulfazi.
Degradation of idursulfase is achieved by generally well understood protein hydrolysis mechanisms to produce small peptides and amino acids, consequently renal and liver function impairment is not expected to affect the pharmacokinetics of idursulfase.
Po razkroju idursulfaze, ki poteka po splošno dobro znanih mehanizmih hidrolize beljakovin, nastanejo majhni peptidi in aminokisline, zato se ne pričakuje, da bi okvara delovanja ledvic ali jeter vplivala na farmakokinetiko idursulfaze.
Analyses of genotypes of patients in study HGT-ELA-038
Analize genotipov bolnikov v študiji HGT-ELA-038
Urine GAG levels were normalized below the upper limit of normal (defined as 126.6 µg GAG/mg creatinine) in 50% of the patients receiving weekly treatment.
Ravni GAG v urinu so se pri 50 % bolnikov, ki so prejemali tedensko zdravljenje, normalizirale pod zgornjo mejo normalne vrednosti (opredeljene kot 126,6 µg GAG/mg kreatinina).
The most common were infusion-related reactions, 202 of which were reported in 22 out of 32 patients following administration of a total of 1580 infusions.
Med njimi so o 202 učinkih poročali pri 22 od 32 bolnikov, po infundiranju skupno 1580 infuzij.
In a 52-week, randomized, double-blind, placebo-controlled clinical study, 96 patients between the ages of 5 and 31 years received Elaprase 0.5 mg/kg every week (n=32) or 0.5 mg/kg every other week (n=32), or placebo (n=32).
V 52-tedenski, randomizirani, dvojno slepi, s placebom nadzorovani klinični študiji je 96 bolnikov, starih od 5 do 31 let, prejemalo zdravilo Elaprase, in sicer 0,5 mg/kg vsak teden (n = 32), 0,5 mg/kg vsak drugi teden (n = 32) ali placebo (n = 32).
Study TKT024
Študija TKT024
* idursulfase is produced by recombinant DNA technology in a continuous human cell line.
* Idursulfaza se izdeluje v neprekinjeni humani celični liniji s tehnologijo rekombinantne DNA.
It is supplied in a glass vial as a clear to slightly opalescent, colourless solution.
Na voljo je v stekleni viali v obliki prozorne do rahlo opalescentne brezbarvne raztopine.
Iduronate-2-sulfatase functions to catabolize the glycosaminoglycans (GAG) dermatan sulfate and heparan sulfate by cleavage of oligosaccharide-linked sulfate moieties.
Iduronat-2-sulfataza katabolizira glikozaminoglikanove (GAG) dermatansulfate in heparanove sulfate s cepljenjem oligosaharidno-povezanih sulfatnih delov.
In a post-hoc analysis of immunogenicity in studies TKT024 and TKT024EXT (see section 4.8), patients were shown to have either the mis-sense mutation or the frameshift / nonsense mutation.
V post hoc analizi imunogenosti na podlagi študij TKT024 in TKT024EXT (glejte poglavje 4.8) so pokazali, da imajo bolniki drugačnosmiselno mutacijo ali mutacijo s premikom bralnega okvirja/nesmiselno mutacijo.
• Flushing (redness)
• zardevanje (rdečica)
If you are treated with Elaprase you may experience side effects during or following an infusion (see section 4 Possible side effects).
Če se zdravite z zdravilom Elaprase, boste morda med infundiranjem ali po njem imeli neželene učinke (glejte poglavje 4 Možni neželeni učinki).
52 weeks of treatment 0.5 mg/kg weekly Marginally weighted (OM) mean (SE)
52 tednov zdravljenja 0,5 mg/kg/teden Marginalno opazovana meja (OM) srednja vrednost (SN)
Patients who have experienced anaphylactoid/anaphylactic reactions should be treated with caution when readministering idursulfase, appropriately trained personnel and equipment for emergency resuscitation (including epinephrine) should be available during infusions.
Pri bolnikih, ki so že imeli anafilaktoidne/anafilaktične reakcije, je pri ponovni uporabi idursulfaze potrebna previdnost; med infuzijo morata biti na voljo ustrezno usposobljeno osebje in oprema za nujno oživljanje (vključno z adrenalinom).
This was an open-label, multicenter, single-arm study of idursulfase infusions in male Hunter syndrome patients between the age of 16 months and 7.5 years.
To je bila odprta, multicentrična študija z enim krakom, v kateri so ocenjevali infuzijo idursulfaze pri bolnikih moškega spola s Hunterjevim sindromom, starih od 16 mesecev do 7,5 leta.
Do not use this medicine if you notice that there is discolouration or presence of foreign particles.
Ne uporabljajte tega zdravila, če opazite, da je raztopina spremenila barvo ali so v njej delci.
It is recommended to deliver the total volume of infusion using a 0.2 µm in line filter.
Priporoča se aplikacija skupnega volumna infuzije skozi 0,2 µm linijski filter.