natgillin/translations-raw huggingface dataset:
The safety and efficacy of Myozyme in patients with renal or hepatic insufficiency have not been evaluated and no specific dosage regimen can be recommended for these patients.
Varnost in učinkovitost zdravila Myozyme pri bolnikih z ledvično ali jetrno odpovedjo nista ugotovljeni in zanje ne moremo priporočiti posebnega režima odmerjanja.
2 Patient response to treatment should be routinely evaluated based on a comprehensive evaluation of all clinical manifestations of the disease.
2 Odziv bolnika na zdravljenje je treba redno ocenjevati na osnovi vsestranskega ocenjevanja vseh kliničnih manifestacij bolezni.
Hypersensitivity (anaphylactic reaction) to the active substance or to any of the excipients.
Preobčutljivost (anafilaktična reakcija) za zdravilno učinkovino ali katerokoli pomožno snov.
Life-threatening anaphylactic reactions, including anaphylactic shock, have been observed in infantile and late onset patients during Myozyme infusions (see section 4.8).
Med infundiranjem zdravila Myozyme so pri bolnikih z zgodnjim in poznim nastopom bolezni zabeležili življenjsko nevarne anafilaktične reakcije, vključno z anafilaktičnim šokom (glejte poglavje 4. 8).
Because of the potential for severe infusion associated reactions, appropriate medical support measures should be readily available when Myozyme is administered.
Zaradi možnosti hudih reakcij, povezanih z infuzijo (IAR), mora biti ob uporabi zdravila Myozyme takoj na voljo ustrezna zdravniška pomoč.
If severe or anaphylactic reactions occur, immediate discontinuation of Myozyme infusion should be considered and appropriate medical treatment should be initiated.
Če pride do hudih ali anafilaktičnih reakcij, je treba z infundiranjem zdravila Myozyme takoj prekiniti in začeti z ustreznim zdravljenjem.
Approximately half of the patients treated with Myozyme in infantile onset clinical studies developed infusion associated reactions (IARs), defined as any related adverse event occurring during the infusion or during the hours following infusion.
Pri približno polovici bolnikov, zdravljenih z zdravilom Myozyme v kliničnih študijah zgodnjega nastopa bolezni, so se pojavile reakcije ob infuziji, opredeljene kot katerikoli neželeni učinek, povezan z zdravilom, ki nastopi med infundiranjem ali nekaj ur po infuziji.
Some reactions were severe (see section 4.8).
Nekatere reakcije so bile hude (glejte poglavje 4. 8).
A tendency was observed in infantile patients treated with a higher dose (40 mg/ kg) to experience more symptoms when developing IARs.
Pri bolnikih z zgodnjim nastopom bolezni, zdravljenih z višjim odmerkom (40 mg/ kg), je bilo opaziti več simptomov pri razvoju infuzijskih reakcij.
Infantile onset patients who develop high antibody titres appear to be at higher risk for developing more frequent IARs.
Bolniki z zgodnjim nastopom bolezni, pri katerih se razvijejo visoki titri protiteles, so izpostavljeni večjemu tveganju za razvoj pogostejših infuzijskih reakcij.
Patients with an acute illness (e. g. pneumonia, sepsis) at the time of Myozyme infusion appear to be at greater risk for IARs.
Bolniki z akutno boleznijo (npr. pljučnico, sepso) so v času infuzije zdravila Myozyme izpostavljeni večjemu tveganju za infuzijske reakcije.
Careful consideration should be given to the patient’ s clinical status prior to administration of Myozyme.
Pred uporabo zdravila Myozyme je treba temeljito preučiti bolnikovo klinično stanje.
Patients should be closely monitored and all cases of IARs, delayed reactions and possible immunological reactions should be reported.
Bolnike je treba skrbno opazovati in poročati o vseh primerih infuzijskih reakcij, zapoznelih reakcij in morebitnih imunoloških reakcij.
Patients who have experienced IARs should be treated with caution when re-administering Myozyme (see section 4.3 and 4.8).
Bolnike, pri katerih so se pojavile infuzijske reakcije, je treba pri ponovni uporabi zdravila Myozyme zdraviti previdno (glejte poglavji 4. 3 in 4. 8).
Reduction of the infusion rate, temporary interruption of the infusion, or pre-treatment, generally with oral antihistamine and/ or antipyretics and/ or corticosteroids, has effectively managed most reactions.
Zmanjšanje stopnje infuzije, začasna prekinitev infuzije ali predhodno zdravljenje, običajno s peroralnim antihistaminikom in/ ali antipiretiki in/ ali kortikosteroidi, je učinkovito ublažilo večino reakcij.
IARs may occur at any time during the infusion of Myozyme or generally up to 2 hours after, and are more likely with higher infusion rates.
Infuzijske reakcije se lahko pojavijo kadarkoli med infuzijo zdravila Myozyme ali običajno do 2 uri kasneje in bolj verjetno pri višjih stopnjah infuzije.
In clinical studies, the majority of patients developed IgG antibodies to rhGAA typically within 3 months of treatment.
V kliničnih študijah je pri večini bolnikov prišlo do tvorbe protiteles IgG pri zdravljenju z rhGAA, običajno v treh mesecih zdravljenja.
A tendency was observed for patients treated with a higher dose (40 mg/ kg) to develop higher titers of antibodies.
Pri bolnikih, zdravljenih z višjim odmerkom (40 mg/ kg), je bilo opaziti razvoj višjih titrov protiteles.
There does not appear to be a correlation between the onset of IARs and the time of antibody formation.
Ni videti, da bi obstajala soodvisnost med nastopom infuzijskih reakcij in časom tvorbe protiteles.
A small number of the IgG positive patients evaluated tested positive for inhibitory effects on in vitro testing.
Pri manjšem številu IgG pozitivnih bolnikov, ki so bili ocenjevani, je bil test za zaviralne učinke pri in vitro preizkusih pozitiven.
Due to the rarity of the condition and the limited experience to date, the effect of antibody formation on safety and efficacy is currently not fully established..
Zaradi redkosti stanja in omejenih izkušenj učinek tvorbe protiteles na varnost in učinkovitost trenutno ni povsem dokazan.
The probability of a poor outcome and of developing high and sustained antibody titers appears higher among CRIM-negative patients (Cross Reactive Immunologic Material; patients in whom no endogenous GAA protein was detected by Western blot analysis) than among CRIM-positive patients (patients in whom endogenous GAA protein was detected by Western blot analysis).
Možnost nezadovoljivega izida in nastanka visokih in vzdrževanih titrov protiteles je višja pri CRIM negativnih bolnikih (navzkrižno reaktivni imunološki material, Cross Reactive Immunologic Material; bolniki, pri katerih niso zaznali endogenega proteina GAA z Western blot analizo) kot pri CRIM pozitivnih bolnikih (bolniki, pri katerih so zaznali endogeni protein GAA z Western blot analizo).
However, high and sustained antibody titers also occur in some CRIM-positive patients.
Vendar pa se visoki in vzdrževani titri protiteles pojavljajo tudi pri nekaterih CRIM- pozitivnih bolnikih.
The cause of a poor clinical outcome and of developing high and sustained antibody titers is thought to be multi-factorial.
Domnevajo, da je vzrok za nezadovoljiv klinični izid ter za razvoj visokih in vzdrževanih titrov protiteles skupek več faktorjev.
Antibody titers should be regularly monitored.
Titre protiteles je treba redno nadzorovati.
Transient nephrotic syndrome which resolved following temporary interruption of ERT was observed in one patient with infantile-onset Pompe disease who received very frequent dosing of rhGAA (10 mg/ kg 5 times weekly) over an extended period.
3 Pri enem bolniku z zgodnjim nastopom Pompejeve bolezni in ki je v daljšem časovnem obdobju zelo pogosto prejemal odmerek rhGAA (10 mg/ kg 5- krat na teden), so opazili prehoden nefrotski sindrom, ki je minil po začasni prekinitvi ERT.
3 4.5 Interaction with other medicinal products and other forms of interaction
4. 5 Medsebojno delovanje z drugimi zdravili in druge oblike interakcij
No formal drug-drug interactions studies have been carried out with alglucosidase alfa.
Za alglukozidazo alfa niso izvedli nobene formalne študije o medsebojnem delovanju med zdravili.
Because it is a recombinant human protein, alglucosidase alfa is an unlikely candidate for cytochrome P450 mediated drug-drug interactions.
Ker je alglukozidaza alfa rekombinantna človeška beljakovina, je malo verjetno, da bi imela kakšno medsebojno delovanje z drugimi zdravili, ki se presnavljajo s pomočjo citokroma P450.
Myozyme should not be used during pregnancy unless clearly necessary.
Med nosečnostjo zdravila Myozyme ni dovoljeno uporabljati, razen v nujnih primerih.
In clinical trials, 39 infantile-onset patients were treated with Myozyme for more than three years (168 weeks with a median of 121 weeks; see section 5.1).
V kliničnih preskušanjih so 39 bolnikov z zgodnjim nastopom bolezni zdravili z zdravilom Myozyme več kot tri leta (168 tednov z mediano 121 tednov; glejte poglavje 5. 1).
Most adverse events reported in these 39 patients were due to manifestations of Pompe disease and not related to the administration of Myozyme.
Večina neželenih dogodkov, zabeleženih pri navedenih 39 bolnikih se je pojavila zaradi manifestacij Pompejeve bolezni in ni bila povezana z uporabo zdravila Myozyme.
Adverse Drug Reactions (ADRs) are listed in Table 1 by System Organ Class.
Neželeni učinki (ADR) so navedeni v tabeli 1 po organskih sistemih.
The ADRs listed are reported in more than 1 patient and shown as percentage of patients experiencing the ADR with the absolute number of patients in brackets.
Navedeni neželeni učinki so zabeleženi pri več kot enem bolniku in prikazani kot odstotek bolnikov, pri katerih so se neželeni učinki pojavili, z absolutnim številom bolnikov v oklepajih.
ADRs 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).
Neželeni učinki so bili najpogosteje blagi do zmerni in skoraj vsi so se pojavili v času infuzije ali v dveh urah po prejemu infuzije (z infuzijo povezane reakcije, IAR).
4 Table 1.
4 Tabela 1.
Adverse Drug Reactions by System Organ Class in more than one patient.
Neželeni učinki na zdravilo glede na organske sisteme pri več kot enem bolniku.
Preferred term
Neželeni učinek
Frequency (no. of patients) N = 39
Pogostost (št. bolnikov) n = 39
Psychiatric disorders Nervous system disorders Cardiac disorders
Psihiatrične motnje
Agitation Tremor Tachycardia Cyanosis
Bolezni živčevja
5% (2 pts) 5% (2 pts) 10% (4 pts) 5% (2 pts)
10% (4 bolniki)
Vascular disorders
cianoza zardevanje
Flushing Hypertension Pallor
hipertenzija bledica kašelj
13% (5 pts) 8% (3 pts) 5% (2 pts)
8% (3 bolniki) 5% (2 bolnika) 13% (5 bolnikov)
Respiratory, thoracic and mediastinal Cough disorders
tahipneja bruhanje
13% (5 pts)
13% (5 bolnikov) 10% (4 bolniki)
Tachypnoea Gastrointestinal disorders Vomiting Retching Nausea Skin and subcutaneous tissue Urticaria disorders
bljuvanje navzea urtikarija
13% (5 pts) 10% (4 pts) 5% (2 pts) 5% (2 pts) 18% (7 pts)
5% (2 bolnika) 5% (2 bolnika) 18% (7 bolnikov)
Rash Rash macular Erythema Rash maculo-papular Rash papular Pruritis General disorders and administration Pyrexia site conditions
osip makularni izpuščaj eritem makulopapulozen izpuščaj papulozen izpuščaj pruritis
15% (6 pts) 5% (2 pts) 8% (3 pts) 8% (3 pts) 5% (2 pts) 5% (2 pts) 28% (11 pts)
15% (6 bolnikov) 5% (2 bolnika) 8% (3 bolniki) 8% (3 bolniki) 5% (2 bolnika) 5% (2 bolnika)
Irritability Chills Investigations Oxygen saturation decreased Blood pressure increased Body temperature increased Heart rate increased
povišan krvni tlak zvišana telesna temperatura pospešen srčni utrip
5% (2 pts) 5% (2 pts) 18% (7 pts) 5% (2 pts) 5% (2 pts) 5% (2 pts)
5% (2 bolnika) 5% (2 bolnika) 5% (2 bolnika)
A limited number of patients with late-onset Pompe disease (see section 5.1) have been treated with Myozyme.
Z zdravilom Myozyme so zdravili omejeno število bolnikov s poznim nastopom Pompejeve bolezni (glejte poglavje 5. 1).
ADRs reported in 2 of 9 late-onset patients treated with Myozyme for up to 1 year in 3 different studies included increased heart rate, hypertension, headache, peripheral coldness, paresthesia, flushing, infusion site pain, infusion site reaction, increased blood pressure and dizziness.
Neželeni učinki, poročani pri 2 od 9 bolnikov s poznim nastopom Pompejeve bolezni, zdravljenih z zdravilom Myozyme do 1 leta, v 3 različnih študijah so vključevale povišan srčni utrip, visok krvni tlak, glavobol, periferno hladnost, parestezijo, zardevanje, bolečino na mestu infundiranja, reakcijo na mestu infundiranja, povišan krvni tlak in vrtoglavico.
The 2 late onset patients did not experience the same ADRs.
Pri dveh bolnikih s poznim nastopom bolezni niso zaznali istih neželenih učinkov.
The ADRs were mild in intensity and assessed as an IAR.
Neželeni učinki so bili v blagi obliki in ocenjeni kot IAR.
IARs in patients treated with Myozyme in clinical studies and expanded access programs and reported in more than 1 patient included rash, flushing, urticaria, pyrexia, cough, tachycardia, decreased oxygen saturation, vomiting, tachypnea, agitation, increased blood pressure, cyanosis, hypertension, irritability, pallor, pruritus, retching, chills, tremor, hypotension, heart rate increased, bronchospasm, erythema, face edema, feeling hot, headache, hyperhidrosis, lacrimation increased, livedo reticularis, nausea, periorbital edema, restlessness and wheezing.
Infuzijske reakcije pri bolnikih, zdravljenih z zdravilom Myozyme, v kliničnih študijah in programih z razširjenim dostopom ter zabeležene pri več kot 1 bolniku so vključevale izpuščaje, rdečico, urtikarijo, zvišano telesno temperaturo, kašelj, tahikardijo, zmanjšano saturacijo kisika, bruhanje, tahipnejo, agitacijo, povišan krvni tlak, cianozo, hipertenzijo, razdražljivost, bledico, pruritus, bljuvanje, mrzlico, tremor, hipotenzijo, pospešen srčni utrip, krč sapnic, eritem, obrazni edem, občutek vročice, glavobol, čezmerno znojenje, povečano solzenje, marmorirano obarvanje kože, navzea, periorbitalni edem, nemirnost in sopihanje.
Those IARs reported as severe in more than 1 patient included pyrexia, decreased oxygen saturation, tachycardia, cyanosis and hypotension.
Infuzijske reakcije, zabeležene kot hude pri več kot 1 bolniku, so vključevale zvišano telesno temperaturo, zmanjšano saturacijo kisika, tahikardijo, cianozo in hipotenzijo.
A single event of angioneurotic edema of severe intensity has been reported.
Zabeležen je bil le en primer močnega angionevrotičnega edema.
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.
V kliničnih preskušanjih in ob komercialni uporabi je pri majhnem številu bolnikov (< 1%) prišlo do anafilaktičnega šoka in/ ali zaustavitve delovanja srca med infundiranjem zdravila Myozyme, kar je zahtevalo izvajanje postopkov oživljanja.
Patients
Pri bolnikih se
5 with moderate to severe or recurrent IARs have been evaluated for Myozyme specific IgE antibodies; some patients tested positive including one patient who experienced an anaphylactic reaction.
Bolnike z zmerno do hudo obliko infuzijskih reakcij ali rekurentnimi infuzijskimi reakcijami so ocenjevali zaradi protiteles IgE, značilnih za zdravilo Myozyme; pri nekaterih bolnikih je bil test pozitiven, vključno z bolnikom, pri katerem je prišlo do anafilaktične reakcije.
In clinical studies doses up to 40 mg/ kg body weight were used.
V kliničnih študijah so bili uporabljeni odmerki do 40 mg/ kg telesne mase.
Alimentary tract and metabolism products – enzymes.
Zdravila za bolezni prebavil in presnove – encimi.
A16AB07 alglucosidase alfa.
A16AB07 alglukozidaza alfa.
Alglucosidase alfa is a recombinant form of human acid α -glucosidase and is produced by recombinant DNA technology using Chinese Hamster Ovary (CHO) cell culture.
Alglukozidaza alfa je rekombinantna oblika človeške kisline α- glukozidaze, ki se pridobiva s tehnologijo rekombinantne DNK iz celične kulture jajčnika kitajskega hrčka (Chinese Hamster Ovary, CHO).
Pompe disease Pompe disease is a rare, progressive and fatal metabolic myopathy with an estimated global incidence of 1 in 40,000 births.
Pompejeva bolezen Pompejeva bolezen je redka, progresivna in smrtna metabolična miopatija s predvidenim globalnim pojavom 1 primera na 40. 000 rojstev.
Other names for Pompe disease include glycogen storage disease type II (GSD- II), acid maltase deficiency (AMD) and glycogenosis type II.
Druga imena za Pompejevo bolezen so bolezen kopičenja glikogena tipa II (GSD- II), pomanjkanje kisline maltaze (AMD) in glikogenoza tipa II.
Pompe disease belongs to the lysosomal storage disorders as it is caused by a deficiency of a naturally-occurring lysosomal hydrolase, acid α - glucosidase (GAA) that degrades lysosomal glycogen to glucose.
Pompejeva bolezen spada med motnje lizosomskega shranjevanja, ki jih povzroči pomanjkanje naravne lizosomske hidrolaze, kisline α- glukozidaze (GAA), ki razgradi lizosomski glikogen do glukoze.
The clinical presentation of Pompe disease can be described as a spectrum of disease which ranges from a rapidly-progressing infantile-onset form (onset of symptoms of Pompe disease typically within the first year of life and a very short expected life-span) to a less rapidly-progressing late-onset form.
Klinično predstavitev Pompejeve bolezni je mogoče opisati kot spekter bolezni, ki sega od hitro - napredujoče oblike z zgodnjim nastopom (nastop simptomov Pompejeve bolezni običajno v prvem letu življenja in zelo kratka pričakovana življenjska doba), do manj hitro- napredujoče oblike s poznim nastopom.
Motor development is often completely arrested, or if motor milestones are achieved, they are subsequently lost.
Razvoj motorike pogosto povsem zastane ali če pa so dosežene popolne motorične sposobnosti, le- te kasneje izginejo.
Death typically occurs due to cardiac and/ or respiratory failure before the age of one year.
Smrt običajno nastopi zaradi srčne in/ ali dihalne odpovedi pred starostjo enega leta.
In a retrospective natural history study in patients with infantile-onset Pompe disease (n=168), the median age at onset of symptoms was 2.0 months and the median age of death was 9.0 months.
V študiji retrospektivne analize podatkov pri bolnikih z zgodnjim nastopom Pompejeve bolezni (n=168) je bila srednja starost ob nastopu simptomov 2, 0 meseca in srednja starost ob smrti 9, 0 mesecev.
Kaplan-Meier survival rates at 12, 24 and 36 months of age were 26%, 9% and 7%, respectively.
Stopnje preživetja po Kaplan- Meierju pri starosti 12, 24 in 36 mesecev so bile 26%, 9% oziroma 7%.
Usually, it is characterised by the presence of sufficient residual GAA activity to preclude the development of cardiomyopathy, however some cardiac involvement has been reported in up to approximately 4% of patients with late-onset Pompe disease.
Ponavadi je zanjo značilna prisotnost zadostne preostale aktivnosti GAA za preprečitev razvoja kardiomiopatije, vendar je bila pri približno 4% bolnikov s poznim nastopom Pompejeve bolezni zabeležena srčna prizadetost.
6 ambulation and respiratory failure, others progressing less rapidly, and yet others presenting with a dissociation in the progression of skeletal and respiratory muscle involvement.
6 Pri bolnikih s poznim nastopom Pompejeve bolezni, ki jo običajno spremlja progresivna miopatija pretežno proksimalnih mišic v medeničnem in ramenskem obroču in različne stopnje dihalne prizadetosti, na koncu stanje napreduje do hude nesposobnosti in/ ali potrebe po podpori respiratorja.
It is postulated that Myozyme will restore lysosomal GAA activity resulting in stabilisation or restoration of cardiac and skeletal muscle function (including respiratory muscles).
Domneva se, da Myozyme ponovno vzpostavi lizosomsko aktivnost GAA, kar privede do uravnoteženja ali obnovitve delovanja srčne in skeletne mišice (vključno z dihalnimi mišicami).
Due to the blood- brain barrier effect and the enzyme’ s size, uptake of alglucosidase alfa in the central nervous system is unlikely.
Zaradi učinka krvno- možganske pregrade in velikosti encima je prehod alglukozidaze alfa v osrednji živčni sistem malo verjeten.
Infantile-onset Pompe disease; clinical trial in patients aged 6 months or less The safety and efficacy of Myozyme was assessed in a pivotal, randomised, open-label, historically- controlled clinical trial of 18 non-ventilated infantile-onset patients aged 6 months or less at the onset of treatment.
Zgodnji nastop Pompejeve bolezni; klinično preskušanje pri bolnikih starih 6 mesecev ali manj Varnost in učinkovitost zdravila Myozyme so ocenili v osrednji, randomizirani, odprti, sledeni klinični študiji 18 bolnikov brez predihavanja z zgodnjim nastopom bolezni, starih 6 mesecev ali manj ob začetku zdravljenja.
Patients were randomized to receive either 20 mg/ kg or 40 mg/ kg every two weeks for a period of 52 weeks.
Bolniki so bili naključno izbrani za prejemanje 20 mg/ kg ali 40 mg/ kg vsaka dva tedna v obdobju 52 tednov.
After 52 weeks of treatment, all 18 patients treated with Myozyme were alive and 15 of these 18 patients were alive and free of invasive ventilatory support whereas 1 of 42 patients in the untreated historical cohort was alive at 18 months of age.
Po 52 tednih zdravljenja je bilo vseh 18 bolnikov, zdravljenih z zdravilom Myozyme, živih in 15 od teh ni potrebovalo podpore invazivnega predihavanja, medtem ko je bil 1od 42 bolnikov iz nezdravljene historične kohorte živ pri 18 mesecih.
7 Table 2:
Tabela 2:
Results for endpoint survival using the Cox regression model
Rezultati preživetja po Coxovem regresijskem modelu Razmerje
Historical
Historična
Treatment
med učinkom
Treated
Zdravljeni referenčna
Reference
in tveganostjo
Effect Hazard Confidence
interval
Patients
bolniki primerjava
Comparator
Cilj
Endpoint
zdravljenja
Ratio Interval
zaupanja
N=18
n=42
N=42
n=18
Results are from a Cox proportional hazards regression analysis which includes treatment as a time-varying covariate, and also includes age of diagnosis and age at symptom onset.
Rezultati so iz Coxove regresijske analize sorazmernih tveganj, ki vključuje zdravljenje kot časovno variirajočo kovariablo in tudi starost diagnoze in starost ob nastopu simptomov.
Echocardiographic indices of cardiomyopathy improved as measured by a decrease in left ventricular mass (LVM).
7 Ehokardiografski indeksi kardiomiopatije so se izboljšali, kot je ugotovljeno z upadom mase levega prekata (LVM).
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.
Po 52 tednih zdravljenja je 14 izmed 18 bolnikov (77, 8%) ohranilo ali izboljšalo odstotek telesne mase glede na starost (nad 3 odstotke), 14 izmed 15 bolnikov (93, 3%) je bilo nad 3 odstotke glede na višino ter 12 izmed 15 bolnikov (80, 0%) je bilo nad 3 odstotke glede na obseg glave.
In the second year of treatment, 15 out of 17 patients had further improved weight-for-age percentiles (with individual patient treatment durations ranging from 78 to 142 weeks; mean follow-up period of 111 weeks), 10 out of 16 patients had further improved length-for-age percentiles (with individual patient treatment durations ranging from 90 to 130 weeks; mean follow-up period of 113 weeks) and 11 out of 15 patients had further improved head circumference-for-age percentiles (with individual patient treatment durations ranging from 90 to 130 weeks; mean follow-up period of 110 weeks).
V drugem letu zdravljenja je 15 izmed 17 bolnikov še nadalje izboljšalo odstotek telesne mase glede na starost (s trajanjem zdravljenja pri posameznih bolnikih v razponu od 78 do 142 tednov; povprečno obdobje sledenja 111 tednov), 10 izmed 16 bolnikov je še nadalje izboljšalo odstotek višine glede na starost (s trajanjem zdravljenja pri posameznih bolnikih v razponu od 90 do 130 tednov; povprečno obdobje sledenja 113 tednov) ter 11 izmed 15 bolnikov je še nadalje izboljšalo odstotek obsega glave glede na starost (s trajanjem zdravljenja pri posameznih bolnikih v razponu od 90 do 130 tednov; povprečno obdobje sledenje 110 tednov).
Based on these results the 20 mg/ kg qow dose is recommended.
Glede na te rezultate je priporočen odmerek 20 mg/ kg.