natgillin/translations-raw huggingface dataset:
Only filters intended for use with transfusion sets should be used to prevent inadvertent removal of cells from the product.
Uporabiti je dovoljeno le filtre, namenjene za uporabo s transfuzijskimi kompleti, da bi preprečili nenamerno odstranitev celic iz zdravila.
Local biosafety guidelines applicable for such products should be followed (see section 6.6).
Upoštevati je treba lokalne smernice o biološki varnosti, ki veljajo za takšna zdravila (glejte poglavje 6.6).
With this condition the immune system does not work properly to defend the body against infections.
Gre za motnjo, pri kateri imunski sistem ne deluje pravilno za obrambo telesa pred okužbami.
This should be harvested from the patient at least 3 weeks prior to treatment with Strimvelis.
Ta vzorec je treba bolnikom odvzeti vsaj 3 tedne pred zdravljenjem z zdravilom Strimvelis.
Busulfan plasma levels should be measured after the first dose of each day by serial blood sampling using an appropriate method.
Koncentracijo busulfana v plazmi je treba izmeriti vsak dan po prvem odmerku z zaporednim vzorčenjem krvi in uporabo ustrezne metode.
It can only be given in a specialised hospital, and by a doctor who is experienced in treating patients with ADA-SCID and in using this type of medicine.
Uporabi se lahko le v specializirani bolnišnici in dati ga sme le zdravnik, ki ima izkušnje z zdravljenjem bolnikov z ADA-SCID in uporabo tovrstnih zdravil.
Non-immunological manifestations of ADA-SCID may not respond to Strimvelis.
Neimunološke manifestacije ADA-SCID se lahko ne odzovejo na zdravilo Strimvelis.
The total busulfan dose is 4 mg/kg, divided into 8 doses of 0.5 mg/kg.
Celotni odmerek busulfana je 4 mg/kg, razdeljen na 8 odmerkov po 0,5 mg/kg.
Healthcare professionals handling Strimvelis should therefore take appropriate precautions to avoid potential transmission of infectious diseases.
Zdravstveni delavci, ki imajo opravka z zdravilom Strimvelis, morajo zato upoštevati ustrezne previdnostne ukrepe za preprečitev prenosa infekcijskih bolezni.
In such cases, the doctor will give you (or your child) replacement stem cells, using the backup sample that was collected and stored before treatment started (see also section 3, How Strimvelis is given).
V takšnem primeru boste (oz. bo vaš otrok) dobili nadomestne matične celice iz rezervnega vzorca, ki je bil odvzet in shranjen pred začetkom zdravljenja (glejte tudi 3. poglavje, Kako se daje zdravilo Strimvelis).
Median values at 3 years post gene therapy were below the normal range.
Mediani vrednosti 3 leta po genskem zdravljenju sta bili pod normalnim območjem.
Strimvelis should not be used during pregnancy.
Zdravila Strimvelis se ne sme uporabljati med nosečnostjo.
If the product contains less than 2 million CD34+ cells/kg, the treating physician should make a decision whether to proceed with administration, based on an individual benefit risk assessment.
Če zdravilo vsebuje manj kot 2 milijona celic CD34+/kg, se mora lečeči zdravnik odločiti, ali bo nadaljeval z dajanjem, upoštevaje individualno oceno koristi in tveganja.
Elderly Strimvelis is not intended for use in patients >65 years of age, and has not been studied in this age group.
Zdravilo Strimvelis ni namenjeno za uporabo pri bolnikih, starejših od 65 let, in v tej starostni skupini ni raziskano.
In clinical trials of gene therapy for other diseases (not ADA-SCID), some patients developed leukaemia or other cancers of the blood system.
V kliničnih preskušanjih genskega zdravljenja drugih bolezni (ne ADA-SCID) so se nekaterim bolnikom pojavili levkemija ali druge vrste rakov krvnega sistema.
• decreases in the number of red or white blood cells (anaemia, neutropenia)*
• zmanjšano število rdečih ali belih krvnih celic (anemija, nevtropenija)*
dAXP results are based on a responder analysis of the percentage of patients following gene therapy who met the definition of adequate metabolic detoxification, therefore baseline value is not applicable.
Rezultati dAXP temeljijo na analizi odzivnega odstotka bolnikov, ki so po genskem zdravljenju izpolnjevali definicijo ustrezne presnovne detoksifikacije, zato izhodiščna vrednost ne pride v poštev.
At least 3 weeks before treatment
Vsaj 3 tedne pred zdravljenjem
b Based on the 3 year post-treatment period for survival and severe infections, and the data collected at the 3 year visit for T-lymphocytes and dAXP.
b Na podlagi 3-letnega obdobja po zdravljenju za preživetje in hude okužbe ter podatke, zbrane na obisku po 3 letih za limfocite T in dAXP.
This medicine contains approximately 3.5 mg sodium per millilitre.
To zdravilo vsebuje približno 3,5 mg natrija na mililiter.
Strimvelis is intended for autologous use only (see section 4.4).
Zdravilo Strimvelis je namenjeno le za avtologno uporabo (glejte poglavje 4.4).
If clinically relevant quality issues, such as out of specification results, are identified after Strimvelis has been infused, the treating physician will be notified.
Če bodo po infundiranju zdravila Strimvelis ugotovljeni klinično pomembni problemi s kakovostjo, na primer rezultati zunaj specifikacij, bo lečeči zdravnik o tem obveščen.
Patients should be closely monitored for the occurrence of severe and opportunistic infections, immune reconstitution parameters and the need for replacement intravenous immunoglobulin (IVIG); in case of lack of response, it is recommended to introduce other ADA-SCID treatments under the supervision of a physician.
Bolnike je treba natančno nadzorovati glede hudih in oportunističnih okužb, parametrov imunske obnove in potrebe po nadomestnem intravenskem imunoglobulinu (IVIG); če ni odziva, je priporočljivo pod zdravniškim nadzorstvom uvesti druge načine zdravljenja ADA-SCID.
No. of infusion bags:
Št. infuzijskih vrečk:
Hypersensitivity to the product or to any of the excipients listed in section 6.1.
Preobčutljivost na zdravilo ali katero koli pomožno snov, navedeno v poglavju 6.1.
n/a - not applicable.
n/a - navedba smiselno ni potrebna.
All the identified adverse reactions in the table (apart from those potentially related to busulfan) are considered to be related to immune reconstitution, due to their nature and timing.
Vsi ugotovljeni neželeni učinki, navedeni v preglednici (razen tistih, ki so morda povezani z busulfanom), zaradi svoje narave in časa pojava veljajo za povezane z imunsko obnovo.
3 days and 2 days before treatment
3 dni in 2 dni pred zdravljenjem
Local biosafety guidelines applicable should be followed (see section 4.2).
Upoštevati je treba lokalne smernice o biološki varnosti (glejte poglavje 4.2).
Low levels of human cells and vector sequences were also detected in non-haematopoietic organs consistent with the presence of blood containing transduced human cells.
Nizko raven človeških celičnih in vektorskih zaporedij so odkrili tudi v nehematopoetskih organih, skladno s prisotnostjo krvi, v kateri so transducirane človeške celice.
This medicine contains genetically-modified cells.
To zdravilo vsebuje genetsko spremenjene celice.
• red or purple dots on the skin, bleeding under the skin (immune thrombocytopenic purpura)
• rdeče ali vijoličaste pike na koži, krvavitev pod kožo (imunska trombocitopenična purpura)
Successful generation of T cells after Strimvelis is also likely to be affected by residual thymic function which can become impaired in older children.
Na uspešno nastajanje celic T po zdravilu Strimvelis verjetno vpliva tudi rezidualno delovanje timusa, ki je lahko pri starejših otrocih okrnjeno.
Patient 8 withdrew from the study before 3 year visit, and thus had no data for T-lymphocytes and dAXP.
Bolnik št. 8 je prenehal sodelovati v študiji pred obiskom ob 3. letu spremljanja in zato zanj ni podatkov o limfocitih T in dAXP.
History of previous gene therapy.
Anamneza predhodnega genskega zdravljenja.
Strimvelis should be administered once only.
Zdravilo Strimvelis je treba uporabiti samo enkrat.
Two samples are collected The doctor will collect two samples of bone marrow stem cells before the planned treatment:
Zdravnik bo pred načrtovanim zdravljenjem vzel dva vzorca matičnih celic kostnega mozga:
The ones marked * may be related to busulfan.
Neželeni učinki, označeni z *, so lahko povezani z busulfanom.
Patient 10 had no baseline value for T-lymphocytes.
Bolnik št. 10 ni imel izhodiščne vrednosti za limfocite T.
A cloudy to clear, colourless to pink dispersion of cells.
Motna do bistra, brezbarvna do rožnata disperzija celic.
Patients treated within the pivotal study
Bolniki, zdravljeni v ključni študiji
The safety of Strimvelis was evaluated in 18 subjects, with a median duration of follow-up of 7 years.
Varnost zdravila Strimvelis so ocenili pri 18 preiskovancih; mediano je spremljanje trajalo 7 let.
The MAH will follow up on the risk of immunogenicity, insertional mutagenesis and oncogenesis as well as hepatic toxicity.
MAH bo spremljal tveganje za imunogenost, insercijsko mutagenezo, onkogenezo in hepatotoksičnost.
Very common > 1/10 Common ≥1/100 to <1/10
≥1/100 do < 1/10
A 4-month biodistribution study was performed in mice.
Pri miših so izvedli 4-mesečno študijo biološke porazdelitve.
Any unused medicinal product or waste material should be disposed of in accordance with local biosafety requirements.
Neuporabljeno zdravilo ali odpadni material zavrzite v skladu z lokalnimi predpisi o biološki varnosti.
One subject treated in a pilot study did not have PEG-ADA re-introduction data, and thus was excluded from the intervention-free survival in the integrated population.
En bolnik zdravljen v pilotski študiji ni imel podatkov o ponovni uvedbi PEG-ADA in tako ni bil vključen v analizo preživetja brez ukrepanja v skupni populaciji.
Four subjects had previously received an unsuccessful stem cell transplant from a haploidentical donor and 15 of 18 subjects had previously received prior enzyme replacement therapy with polyethylene-glycol-modified bovine adenosine deaminase (PEG-ADA).
Štirje bolniki so predhodno opravili neuspešno presaditev matičnih celic haploidentičnega dajalca, 15 od 18 bolnikov pa je predhodno prejemalo encimsko nadomestno zdravljenje s polietilenglikol-modificirano govejo adenozin deaminazo (PEG-ADA).
T-lymphocyte (CD3+) and NK (CD56+) cell counts improved following treatment with Strimvelis.
Število limfocitov T (CD3+) in NK (CD56+) se je po zdravljenju z zdravilom Strimvelis izboljšalo.
Strimvelis is intended solely for autologous use and should never be administered to any patient other than the original CD34+ cell donor.
Zdravilo Strimvelis je namenjeno izključno za avtologno uporabo in ga nikdar ne sme dobiti noben drug bolnik razen prvotnega dajalca celic CD34+.
It works by putting a new gene into stem cells in the bone marrow so they can make ADA.
Deluje z vstavitvijo novega gena v matične celice kostnega mozga, tako da lahko izdelujejo ADA.
It will be used to make the Strimvelis, by putting a new gene into the cells.
Ta vzorec bo uporabljen za izdelavo zdravila Strimvelis z vstavitvijo novega gena v celice.
Strimvelis should be used with caution in patients with hypersensitivity to aminoglycosides or bovine serum albumin.
Zdravilo Strimvelis je treba previdno uporabljati pri bolnikih, preobčutljivih na aminoglikozide ali albumin govejega seruma.
Strimvelis is made specially from the patient's own cells.
Zdravilo Strimvelis je posebej izdelano iz bolnikovih lastnih celic.
The efficacy of Strimvelis was evaluated in a 3-year open-label, prospective study in children who lacked a sibling HLA matched stem cell donor and were either failing to respond adequately to PEG-ADA, were intolerant or did not have access to it.
Učinkovitost zdravila Strimvelis so ocenili v 3-letni odprti, prospektivni študiji pri otrocih, ki med sorojenci niso imeli HLA-usklajenega dajalca matičnih celic ter se na zdravljenje s PEG-ADA niso ustrezno odzvali, ga niso prenesli ali jim to zdravljenje ni bilo dostopno.
It is recommended that patients be monitored long term with at least annual visits for the first eleven years and then at 13 and 15 years post treatment with Strimvelis, to include a complete blood count with differential, biochemistry and thyroid stimulating hormone.
Bolnike je priporočljivo dolgoročno nadzorovati s pregledi vsaj enkrat na leto prvih 11 let in potem 13. in 15. leto po zdravljenju z zdravilom Strimvelis; pregledi morajo obsegati celotno krvno sliko z diferencialno krvno sliko, biokemične preiskave in meritev tiroideo stimulirajočega hormona.
About 4 to 5 days before treatment
Približno 4 do 5 dni pred zdravljenjem
The majority were reported during the 3 month to 3 year follow-up period and resolved, with the exception of hypothyroidism and positive ANA tests.
3 mesecev do 3 let spremljanja in so minili (z izjemo hipotiroidizma in pozitivnih testov ANA).
x 106 METHOD AND ROUTE(S) OF ADMINISTRATION Read the package leaflet before use.
x 106 POSTOPEK IN POT(I) UPORABE ZDRAVILA Pred uporabo preberite priloženo navodilo!
It will be stored, to be given to the patient as replacement stem cells if Strimvelis cannot be given or does not work (see ‘When Strimvelis treatment cannot be completed' in section 2)
Ta vzorec bo shranjen kot vir nadomestnih matičnih celic, če zdravila Strimvelis ni mogoče dati, ali če ne deluje (glejte "Če zdravljenja z zdravilom Strimvelis ni mogoče dokončati" v 2. poglavju).
The quantitative information regarding CD34+ cells/kg and total cells in the product is presented in the labelling for each batch.
Kvantitativne informacije o celicah CD34+/kg in celotni vsebnosti celic v zdravilu so navedene na nalepkah vsake serije.
People with ADA-SCID cannot produce enough of an enzyme called adenosine deaminase (ADA) because the gene to make it is faulty.
Pri osebah z ADA-SCID ne nastaja dovolj encima, imenovanega adenozin deaminaza (ADA), ker je okvarjen gen za nastajanje tega encima.
• raised temperature (pyrexia)
• zvišana telesna temperatura (pireksija)
The concentration is 1-10 million CD34+ cells/ml.
Koncentracija je 1-10 milijonov celic CD34+/ml.
It is not known whether the ingredients of Strimvelis can pass into breast milk.
Ni znano, ali lahko sestavine zdravila Strimvelis preidejo v materino mleko.
PHARMACEUTICAL FORM Dispersion for infusion.
FARMACEVTSKA OBLIKA disperzija za infundiranje
B cell function: All 12 subjects treated in the pivotal study were receiving IVIG therapy at the time of screening, and 7 subjects (58%) had discontinued IVIG use during 0-3 years follow-up after gene therapy.
Delovanje celic B: Vseh 12 bolnikov, ki so se zdravili v ključni študiji, je ob presejanju prejemalo zdravljenje z IVIG in 7 bolnikov (58 %) je v obdobju od 0 do 3 let spremljanja po genskem zdravljenju prenehalo uporabljati IVIG.
Long-term PEG-ADA (exceeding 3 months of continuous duration) was used by three subjects; two of these subjects subsequently received a sibling-matched stem cell transplant and one subject remained on chronic PEG-ADA treatment.
Dolgotrajno uporabo PEG-ADA (neprekinjeno več kot 3 mesece) so potrebovali trije bolniki; dvema od njih so nato presadili matične celice usklajenega sorojenca, en bolnik pa je ostal na kroničnem zdravljenju s
In addition, the allergy related adverse reactions in the table were reported mostly during the 3 month to 3 year follow-up period.
Poleg tega so poročali o alergijskih neželenih učinkih, navedenih v preglednici, večinoma v obdobju od 3 mesecev do
Strimvelis is not expected to interact with the hepatic cytochrome P-450 family of enzymes or drug transporters.
Ni pričakovati, da bi zdravilo Strimvelis medsebojno delovalo z družino jetrnega citokroma P-450 ali s prenašalci zdravil.
There are no data on the effects of Strimvelis on human fertility.
Podatkov o vplivu zdravila Strimvelis na plodnost pri človeku ni.
It is recommended that 0.5 mg/kg intravenous busulfan be administered every 6 hours on two consecutive days starting three days before administration of Strimvelis.
Priporočena je intravenska uporaba busulfana v odmerku 0,5 mg/kg na 6 ur dva dni zapored in z začetkom tri dni pred uporabo zdravila Strimvelis.
Strimvelis is used to treat ADA-SCID when there is no suitable match from a family member to donate stem cells from their bone marrow for a transplant.
Zdravilo Strimvelis se uporablja za zdravljenje ADA-SCID, če noben družinski član ni primeren za darovanje matičnih celic kostnega mozga za presaditev.
Local biosafety guidelines applicable should be followed.
Upoštevati morate lokalne smernice o biološki varnosti.
e At baseline 9 of 11 (82%) patients had dAXP <100 nmol/ml.
e Izhodiščno je imelo 9 od 11 (82 %) bolnikov dAXP < 100 nmol/ml.
67% (12 of 18) of Strimvelis treated patients had either autoimmune antibodies or other manifestations (e.g. autoimmune thrombocytopenia, autoimmune aplastic anaemia, autoimmune hepatitis and Guillain-Barré syndrome) (see section 4.8).
67 % (12 od 18) bolnikov, zdravljenih z zdravilom Strimvelis, je imelo ali avtoimunska protitelesa ali druge manifestacije (npr. avtoimunsko trombocitopenijo, avtoimunsko aplastično anemijo, avtoimunski hepatitis ali Guillain-Barréjev sindrom) (glejte poglavje 4.8).
The median percentages of genetically modified cells in peripheral blood at one year and 3 years after treatment were 28% (range 6%-92%) and 30% (range 8%-101%) of CD19+, and 73% (range 20%-100%) and 67% (range 39%-82%) of CD3+ cells, respectively.
Mediani delež genetsko spremenjenih celic v periferni krvi je bil pri celicah CD19+ po 1 letu 28 % (razpon: od 6 do 92 %) in po 3 letih 30 % (razpon: od 8 do 101 %), pri celicah CD3+ pa po 1 letu 73 % (razpon: od 20 do 100 %) in po 3 letih 67 % (razpon: od 39 do 82 %).
The patient must be able to donate adequate CD34+ cells to deliver the minimum 4 million purified CD34+ cells/kg, required for manufacture of Strimvelis.
Pri bolniku mora biti mogoč odvzem ustreznih celic CD34+ za pridobitev najmanj 4 milijonov prečiščenih celic CD34+/kg, potrebnih za izdelavo zdravila Strimvelis.
→ Talk to your doctor or nurse.
→ se posvetujte z zdravnikom ali medicinsko sestro.
Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)matched related stem cell donor is available (see section 4.2 and section 4.4).
Zdravilo Strimvelis je indicirano za zdravljenje bolnikov s hudo kombinirano imunsko pomanjkljivostjo zaradi pomanjkanja adenozin deaminaze (ADA-SCID), za katere ni na voljo primernega, po humanem levkocitnem antigenu sorodnega (HLA-usklajenega) dajalca matičnih celic (glejte poglavji 4.2 in 4.4).
No data from clinical studies are available regarding overdose of Strimvelis.
Iz kliničnih študij ni na voljo podatkov o prevelikem odmerjanju zdravila Strimvelis.
BATCH NUMBER, DONATION AND PRODUCT CODES Lot:
ŠTEVILKA SERIJE, ENOTNE OZNAKE DAROVANJA IN IZDELKOV Lot:
Strimvelis is intended solely for autologous use.
Zdravilo Strimvelis je namenjeno izključno za avtologno uporabo.
A 100% survival rate was observed for all 12 subjects treated within the pivotal study and also for the 18 subjects in the integrated analysis, with a median follow up duration of approximately 7 years.
Preživetje je bilo 100-odstotno tako pri vseh 12 bolnikih, ki so se zdravili v ključni študiji, kot pri vseh 18 bolnikih v skupni analizi; mediano obdobje spremljanja je trajalo približno 7 let.
UNIQUE IDENTIFIER - HUMAN READABLE DATA Not applicable.
EDINSTVENI IDENTIFIKATOR - ZA ČLOVEKA BERLJIVI PODATKI
Rate of severe infections per person year * Excluding data from one patient collected from the time of intervention with PEG-ADA (>3 months treatment) or hematopoietic stem cell transplantation.
Delež hudih okužb na oseboleto * Izključujoč podatke enega bolnika, zbrane od časa ukrepanja s PEG-ADA (> 3 mesece zdravljenja) ali presaditvijo hematopoetskih matičnih celic.
Before infusion, it must be confirmed that the patient's identity matches the essential unique patient information on the Strimvelis infusion bag(s) and/or container (see sections 4.4 and 6.6).
Pred infundiranjem je treba potrditi, da se bolnikova identiteta ujema s ključnimi edinstvenimi bolnikovimi informacijami na infuzijski vrečki/infuzijskih vrečkah zdravila Strimvelis in/ali zunanjem vsebniku (glejte poglavje 4.4 in 6.6).
Unused medicine must be disposed of in compliance with the local biosafety guidelines.
Neporabljeno zdravilo zavrzite v skladu z lokalnimi predpisi za biološko varnost.
These autoimmune adverse reactions were reported for subjects post-gene therapy.
O teh avtoimunskih neželenih učinkih so poročali pri osebah po genskem zdravljenju.
Retroviral insertion sites (RIS) have been detected adjacent to or within CCND2 and LMO2 and there is a potential risk of leukaemic transformation following treatment with Strimvelis.
Znotraj ali ob CCND2 in LMO2 so bila ugotovljena retrovirusna insercijska mesta (RIS - retroviral insertion sites) in po zdravljenju z zdravilom Strimvelis obstaja možno tveganje za levkemično transformacijo.
All patients received busulfan conditioning prior to gene therapy, with most receiving a total dose of 4 mg/kg intravenously over 2 consecutive days prior to CD34+ infusion.
Vsi bolniki so pred genskim zdravljenjem opravili predpripravo z busulfanom; večina jih je prejela skupen odmerek 4 mg/kg intravensko 2 zaporedna dneva pred infundiranjem CD34+).
A transfusion administration set with filter should be used.
Uporabiti je treba komplet za dajanje transfuzije s filtrom.
• wheezing, difficulty breathing (asthma)
• piskajoče ali težko dihanje (astma)
There were no adverse effects on survival, haematological parameters or histopathology of major organs, apart from body weight loss and atrophy in the testes and ovaries consistent with administration of busulfan.
Neželenih učinkov na preživetje, hematološke parametre in histopatologijo pomembnih organov ni bilo, razen zmanjšanja telesne mase in atrofije mod in jajčnikov, skladno z uporabo busulfana.
The MAH shall plan to include regular progress reports of the registry in the PSUR and provide interim study reports every 2 years until the registry finishes.
načrtoval vključitev rednih poročil o napredku registrske študije in bo o njej posredoval vmesna poročila vsaki
A total of 18 patients with ADA-SCID were treated with Strimvelis as part of one open-label pivotal trial (AD1115611; N=12), two early open-label pilot studies (AD1117054/AD1117056; N=3), and a compassionate use program (AD1117064; N=3).
Skupno število bolnikov z ADA-SCID, zdravljenih z zdravilom Strimvelis, je bilo 18: 12 v enem odprtem ključnem preskušanju (AD1115611), 3 v dveh zgodnjih odprtih pilotskih študijah (AD1117054/AD1117056) in 3 v programu sočutne uporabe (AD1117064).
The stem cell back-up is collected for use as rescue treatment should there be a failure during product manufacture, transplant failure, or prolonged bone marrow aplasia after treatment with Strimvelis.
Rezervni vzorec matičnih celic je potreben za rešilno zdravljenje v primeru neuspešne izdelave zdravila, neuspešne presaditve ali dolgotrajne aplazije kostnega mozga po zdravljenju z zdravilom Strimvelis.
T-lymphocyte (x106/l) n median (range) % subjects with venous RBC dAXP <100 nmol/ml after Strimvelisd n
Limfociti T (x106/l) n mediana (razpon) % bolnikov z venskim RBC dAXP < 100 nmol/ml po zdravljenju z zdravilom Strimvelis n
T cell function: In the patients treated in the pivotal study, T cell proliferation was demonstrated in response to stimulation with anti-CD3 antibodies (median 62629 cpm, range 4531 to 252173) and phytohemaglutinin (median 140642 cpm, range 11119 to 505607) at 1 year post gene therapy, and these responses were sustained through Year 3.
Delovanje celic T: Pri bolnikih, zdravljenih v ključni študiji, je bila dokazana proliferacija celic T v odziv na stimulacijo s protitelesi proti CD3 (mediana 62629 cpm, razpon od 4531 do 252173) in fitohemaglutininom (mediana 140642 cpm, razpon od 11119 do 505607) po 1 letu po genskem zdravljenju; ta odziva sta se ohranila do 3. leta.
A completed product consent form is required prior to initiating treatment.
Pred uvedbo zdravljenja mora biti izpolnjen obrazec o soglasju za zdravilo.