natgillin/translations-raw huggingface dataset:
C-reactive protein increased, Aspergillus test positive, Blood potassium decreased, Weight decreased, Blood alkaline phosphatase decreased, Blood magnesium decreased, Forced expiratory flow decreased, Protein total decreased, Blood albumin decreased, Reticulocyte count decreased, Reticulocyte percentage decreased
Preiskave zvišana vrednost C-reaktivnega proteina, pozitiven test na Aspergillus, zmanjšana raven kalija v krvi, zmanjšanje telesne mase, zmanjšana raven alkalne fosfataze v krvi, zmanjšana raven magnezija v krvi, zmanjšan pretok forsiranega zraka med izdihom, zmanjšana raven skupnih beljakovin, zmanjšana raven albumina v krvi, zmanjšano število retikulocitov, zmanjšan odstotek retikulocitov
• Access the medicinal product infusion bag and infuse per the administration site's standard procedures for administration of cell therapy products.
• Vzemite infuzijsko vrečko z zdravilom in jo infundirajte v skladu s standardnimi postopki na mesta za dajanje zdravil pri celičnem zdravljenju.
This medicine contains 391-1564 mg sodium (main component of cooking/table salt) in each dose.
Vsak odmerek zdravila vsebuje 391-1564 mg natrija (glavna sestavina kuhinjske soli).
All patients were administered G-CSF and plerixafor to mobilise stem cells prior to the apheresis procedure.
Vsi bolniki so pred postopkom afereze prejeli G-CSF in pleriksafor za mobilizacijo matičnih celic.
It is unknown whether Zynteglo transduced cells have the potential to be transferred in utero to a foetus.
Ni znano, ali imajo transducirane celice zdravila Zynteglo možnost, da se v maternici prenašajo na plod.
Name and address of the manufacturer(s) responsible for batch release apceth Biopharma GmbH Haidgraben 5
NEMČIJA Ime in naslov izdelovalca (izdelovalcev), odgovornega (odgovornih) za sproščanje serij apceth Biopharma GmbH Haidgraben 5
o Treatment with Zynteglo is associated with the risk of delayed platelet engraftment that could lead to an increased tendency for bleeding.
o Zdravljenje z zdravilom Zynteglo je povezano s tveganjem zapoznele vsaditve trombocitov, kar lahko poveča nagnjenost h krvavitvam.
Zynteglo is given by a drip (infusion) into a vein.
Zdravilo Zynteglo se daje s kapljanjem (infuzijo) v veno.
Bleeding is a potential complication of thrombocytopenia subsequent to myeloablative conditioning and treatment with Zynteglo.
Krvavitev je možni zaplet trombocitopenije, ki sledi mieloablativnem postopku in zdravljenju z zdravilom Zynteglo.
• stomach discomfort with nausea and vomiting
• neugodje v želodcu s slabostjo in bruhanjem
Blood stem cells are collected To make Zynteglo and to serve as replacement cells if needed.
Za izdelavo zdravila Zynteglo in uporabo za nadomestne celice, če bodo potrebne
- The active substance of Zynteglo consists of your own blood stem cells that contain functional copies of the beta-globin gene that can be measured in your blood.
- Učinkovina zdravila Zynteglo je sestavljena iz vaših krvotvornih matičnih celic, ki vsebujejo funkcionalne kopije gena za beta globin, ki ga je mogoče v vaši krvi izmeriti.
Before infusion, it must be confirmed that the patient's identity matches the unique patient information on the Zynteglo infusion bag(s).
Pred infuzijo je treba potrditi, da se identiteta bolnika ujema z edinstvenimi informacijami o bolniku na infuzijski(h) vrečki(vrečkah).
Zynteglo is an autologous gene therapy medicinal product consisting of autologous cells that have been genetically modified ex vivo.
Zdravilo Zynteglo je zdravilo za avtologno gensko terapijo, sestavljeno iz avtolognih celic, ki so bile gensko spremenjene ex vivo.
The treating physician should confirm that HSC transplantation is appropriate for the patient before myeloablative conditioning is initiated (see section 4.4).
Lečeči zdravnik mora potrditi, da je presaditev HSC primerna za bolnika, preden se začne mieloablativni postopek (glejte poglavje 4.4).
It is not known whether the ingredients of Zynteglo can pass into breast milk.
Ni znano, ali lahko sestavine zdravila Zynteglo prehajajo v materino mleko.
No data from clinical studies are available regarding overdose of Zynteglo.
Podatki iz kliničnih študij glede prevelikega odmerjanja zdravila Zynteglo niso na voljo.
Five patients are evaluable for assessment of TI. Of these, 4/5 (80.0%, 95% CI 28.4-99.5%) achieved TI at last follow-up.
Štirje od petih (80,0 %, 95-odstotni interval zaupanja 28,4-99,5 %) so dosegli TI pri zadnjem spremljanju.
Patients not receiving prophylaxis for VOD appeared to be at an increased risk for developing VOD.
Kaže, da je pri bolnikih, ki niso prejeli profilakse za jetrno vensko-okluzivno bolezen, večje tveganje za razvoj jetrne vensko-okluzivne bolezni.
In the 3 patients who did not achieve TI, reductions of 100%, 86.9% and 26.8% in transfusion volume requirements and of 100%, 85.3% and 20.7% in transfusion frequency were observed between Month 6 through Month 24 visit when compared to their pre-study levels of RBC transfusions.
Pri 3 bolnikih, ki niso dosegli TI, so na pregledih od 6. do 24. meseca opazili zmanjšanje potrebne količine transfuzije za 100 %, 86,9 % in 26,8 % ter pogostnosti transfuzij za 100 %, 85,3 % in 20,7 % v primerjavi z njihovimi transfuzijami RBC pred študijo.
Bone marrow biopsies taken before treatment were consistent with a diagnosis of TDT, including a low myeloid/erythroid ratio (N=15, HGB-207), reflective of erythroid hyperplasia.
Biopsije kostnega mozga, odvzete pred zdravljenjem, so bile skladne z diagnozo TDT, vključno z nizkim razmerjem mieloid/eritroid (N=15, HGB-207), kar kaže na eritroidno hiperplazijo.
HSC transplantation must be appropriate for a patient with TDT to be treated with Zynteglo (see section 4.4).
Presaditev celic HSC mora biti primerna za bolnika s TDT, ki bo zdravljen z zdravilom Zynteglo (glejte poglavje 4.4).
Other medicines and Zynteglo
Druga zdravila in zdravilo Zynteglo
Most side effects occur during or within a few days after treatment with Zynteglo but can occur later.
Večina neželenih učinkov se pojavi v nekaj dneh po zdravljenju z zdravilom Zynteglo, vendar se lahko pojavijo tudi pozneje.
MRI of the liver should be performed on all patients prior to myeloablative conditioning.
Magnetno resonančno slikanje (MRI) jeter je treba izvesti pri vseh bolnikih, ki so pred mieloablativnim postopkom.
(Second Infusion
(Druga infuzijska vrečka)
• bleeding or bruising
• krvavitve ali modrice
• The Guide for healthcare professionals shall contain the following key elements: o Warnings and precautions of the mobilisation agents and the myeloablative conditioning agent must be considered.
• Navodilo za zdravstvene delavce mora vsebovati naslednje ključne elemente: o Upoštevati je treba opozorila in previdnostne ukrepe za mobilizacijske učinkovine in učinkovino za mieloablativni postopek.
Mobilisation and apheresis
Mobilizacija in afereza
• pain in back, bone, skin, limbs, anus, or muscles
• bolečine v hrbtu, kosteh, na koži, v okončinah, anusu ali mišicah
Manufacturer apceth Biopharma GmbH Haidgraben 5
Izdelovalec apceth Biopharma GmbH Haidgraben 5
Zynteglo adds functional copies of a modified β-globin gene into the patients' HSCs through transduction of autologous CD34+ cells with BB305 LVV, thereby addressing the underlying genetic cause of the disease.
Zdravilo Zynteglo doda funkcionalne kopije spremenjenega gena za β-globin v bolnikove celice HSC s transdukcijo avtolognih celic CD34+ z BB305 LVV, s čimer se osredotoči na genski vzrok bolezni.
• dark patches on skin
• temne lise na koži
A back-up collection of CD34+ stem cells of at least 1.5 × 106 CD34+ cells/kg (if collected by apheresis) or >1.0 × 108 TNC/kg (if collected by bone marrow harvest) is required.
Potrebno je dodatno zbiranje matičnih celic CD34+, in sicer vsaj 1,5 × 106 celic CD34+ /kg (če jih zbiramo z aferezo) ali > 1,0 × 108 TNC/kg (če jih zbiramo z jemanjem kostnega mozga).
This being a conditional marketing authorisation and pursuant to Article 14(7) of Regulation (EC) No 726/2004, the MAH shall complete, within the stated timeframe, the following measures:
726/2004, mora imetnik dovoljenja za promet z zdravilom v določenem časovnem okviru izvesti naslednje ukrepe:
• unusual hair loss or thinning
• neobičajno izpadanje ali tanjšanje las
Prior to launch of Zynteglo in each Member State the Marketing Authorisation Holder (MAH) must agree about the content and format of the educational and controlled distribution programme,
Pred prihodom zdravila Zynteglo na trg v vsaki državi članici se mora imetnik dovoljenja za promet z zdravilom s pristojnim nacionalnim organom dogovoriti o obliki in vsebini izobraževalnega programa in programa nadzorovane distribucije, vključno s sredstvi obveščanja, načini razdeljevanja in drugimi vidiki programa.
What Zynteglo looks like and contents of the pack
Izgled zdravila Zynteglo in vsebina pakiranja
Patients who experience neutrophil engraftment failure should receive rescue treatment with the back-up collection (see section 4.2).
Bolniki z neuspešno vsaditvijo nevtrofilcev morajo prejeti rešilno zdravljenje z dodatno zbranimi matičnimi celicami (glejte poglavje 4.2).
To prepare your bone marrow for treatment with Zynteglo.
Za pripravo kostnega mozga na zdravljenje z zdravilom Zynteglo
Expiry Date
Do datuma
The concentration is 1.2-20 × 106 CD34+ cells (blood stem cells) per millilitre.
Koncentracija znaša 1,2-20 × 106 celic CD34+ (krvotvornih matičnih celic) na mililiter.
• Do not sample, alter, or irradiate the medicinal product.
• Zdravila ne smete vzorčiti, spremeniti ali obsevati.
Patients should be assessed for hepatic impairment to ensure HSC transplantation is appropriate (see section 4.4.).
Bolnike je treba oceniti za okvaro jeter, da se potrdi primernost presaditve HSC (glejte poglavje 4.4).
Your name and date of birth, as well as coded information identifying you as the patient, are printed onto each infusion bag and each metal container.
Na vsako infuzijsko vrečko in vsak kovinski vsebnik so natisnjeni vaše ime in datum rojstva ter kodirane informacije, ki vas identificirajo kot bolnika.
Anti-retroviral and hydroxyurea use
Uporaba protiretrovirusnih zdravil in hidroksisečnine
• Administer each infusion bag of Zynteglo via intravenous infusion over a period of less than 30 minutes.
• Vsako infuzijsko vrečko zdravila Zynteglo je treba dati kot intravensko infuzijo, ki naj traja manj kot 30 minut.
Standard procedures for patient management after HSC transplantation should be followed after Zynteglo infusion.
Po infundiranju zdravila Zynteglo je treba upoštevati standardne postopke za obravnavo bolnikov po presaditvi HSC.
Transfusion-dependent β-thalassaemia (TDT)
Beta talasemija, ki zahteva transfuzijo (TDT)
The median (min, max) busulfan dose was 3.50 (2.5, 5.0) mg/kg/day (N=32).
odmerka busulfana je bila 3,50 (2,5; 5,0) mg/kg/dan (N=32).
In order to confirm the efficacy and safety of Zynteglo in patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β0/β0
Za potrditev učinkovitosti in varnosti zdravila Zynteglo pri bolnikih, starih 12 let in več, z β-talasemijo, odvisno od transfuzije (TDT), ki nimajo genotipa β0/β0,
Protocol submission:
Predložitev protokola: Julij
• Infuse Zynteglo as soon as possible and no more than 4 hours after thawing.
• Infundirajte zdravilo Zynteglo čim prej oziroma ne kasneje kot v 4 urah po odtajanju.
• a low level of blood platelets, which may reduce the ability of blood to clot
• nizka raven trombocitov v krvi, ki lahko zmanjša sposobnost strjevanja krvi
• This medicinal product contains genetically modified human blood cells.
• To zdravilo vsebuje gensko spremenjene človeške krvne celice.
Apheresis material from patients with a positive test for HIV will not be accepted for Zynteglo manufacturing.
Aferezni material bolnikov s pozitivnim testom za HIV ni sprejemljiv za izdelavo zdravila
Autologous CD34+ cells encoding βA-T87Q-globin gene This medicine is subject to additional monitoring.
Avtologne celice CD34+, ki kodirajo gen za βA-T87Q-globin Za to zdravilo se izvaja dodatno spremljanje varnosti.
In order to confirm the efficacy and safety of Zynteglo in patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β0/β0 genotype, the MAH should submit interim and final data on Study HGB-207
Za potrditev učinkovitosti in varnosti zdravila Zynteglo pri bolnikih, starih 12 let in več, z β-talasemijo, odvisno od transfuzije (TDT), ki nimajo genotipa β0/β0, mora imetnik dovoljenja za promet predložiti vmesne in končne podatke za študijo HGB-207
Account for all infusion bags and confirm each infusion bag of Zynteglo is within the expiry date using the accompanying Lot Information Sheet.
Preštejte vse infuzijske vrečke in potrdite, da so vse infuzijske vrečke zdravila Zynteglo znotraj datuma izteka roka uporabnosti, ki je naveden v priloženem dokumentu za informacije o seriji.
3521AZ Utrecht The Netherlands medinfo@bluebirdbio.com
3521AZ Utrecht Nizozemska medinfo@bluebirdbio.com
• enlarged liver
• povečana jetra
Infuse as soon as possible and no more than 4 hours after thawing.
Infundirajte ga čim prej oziroma najkasneje v 4 urah po odtajanju.
At 48 months after infusion of Zynteglo for patients who achieved TI, the median reduction (min, max) in serum ferritin levels from baseline was 75.02% (39.2, 84.8) (N=3, HGB-204; N=2, HGB-205).
48 mesecev po infundiranju zdravila Zynteglo bolnikom, ki so dosegli TI, je bila mediana zmanjšanja (min, maks) ravni feritina v serumu od izhodišča 75,02 % (39,2; 84,8) (N=3, HGB-204; N=2, HGB-205).
o Package leaflet o A patient/carer guide o A patient alert card
o Navodilo za uporabo o Vodnik za bolnika/skrbnika o Opozorilno kartico za bolnika
• small tear in tissue that lines the anus
• manjša raztrganina tkiva okrog anusa
The minimum recommended dose of Zynteglo is 5.0 × 106 CD34+ cells/kg.
Najmanjši priporočeni odmerek zdravila Zynteglo je 5,0 × 106 celic CD34+ /kg.
• After thaw, mix the medicinal product gently by massaging the infusion bag until all of the contents are uniform.
• Po odtajanju rahlo premešajte zdravilo tako, da infuzijsko vrečko stiskate, dokler vsebina ni enakomerno porazdeljena.
low sodium, low phosphate, decrease in protein, or decrease in albumin
nizko raven magnezija, nizko raven natrija, nizko raven fosfata, znižanje ravni beljakovin ali znižanje ravni albumina
Patients are required to undergo HSC mobilisation followed by apheresis to obtain CD34+ stem cells for medicinal product manufacturing (see section 5.1 for a description of the mobilisation regimen used in clinical studies).
Za pridobitev matičnih celic CD34+ za izdelavo zdravila morajo bolniki opraviti mobilizacijo HSC, ki ji sledi afereza (glejte poglavje 5.1 za opis režima mobilizacije, ki je bil uporabljen v kliničnih študijah).
• The Guide to handling and method of administration for healthcare professionals shall contain the following key elements: o Instructions on receiving and storing of Zynteglo and how to check Zynteglo prior to administration o Instructions about the thawing of Zynteglo o Instructions on protective equipment and treatment of spills.
o Navodila o sprejemu in shranjevanju zdravila Zynteglo in kako je treba zdravilo Zynteglo pred dajanjem pregledati o Navodila o odtajanju zdravila Zynteglo o Navodila o zaščitni opremi in obravnavi razlitja.
The Netherlands e-mail: medinfo@bluebirdbio.com
Nizozemska e-pošta: medinfo@bluebirdbio.com
All patients with non-β0/β0 genotypes received anti-seizure prophylaxis with agents other than phenytoin prior to initiating busulfan.
Vsi bolniki z genotipi, ki ni genotip β0/β0, so bili pred začetkom odmerjanja busulfana deležni protiepileptične profilakse z učinkovinami, ki niso bile fenitoin.
Neutrophil engraftment occurred on median (min, max) Day 19.5 (13, 38) after medicinal product infusion.
prišlo po 19,5 (13, 38) dneh po infundiranju zdravila.
Pre-treatment conditioning
Postopek pred zdravljenjem
After thaw, mix the medicinal product gently by massaging the infusion bag until all of the contents are uniform.
Po odtajanju rahlo premešajte zdravilo tako, da infuzijsko vrečko stiskate, dokler vsebina ni enakomerno porazdeljena.
The Lot Information Sheet is included inside the lid of the cryoshipper used to transport Zynteglo.
Dokument z informacijami o seriji se nahaja v pokrovu kriogenskega transportnega tanka, ki se uporablja za transport zdravila Zynteglo.
The median (min, max) time to last RBC transfusion was 0.95 (0.5, 1.1) months following Zynteglo infusion.
do zadnje transfuzije RBC je bila 0,95 (0,5; 1,1) mesecev po infuziji zdravila Zynteglo.
Women of childbearing potential/Contraception in males and females There are insufficient exposure data to provide a precise recommendation on duration of contraception following treatment with Zynteglo.
Ni dovolj podatkov o izpostavljenosti, da bi zagotovili natančno priporočilo glede trajanja kontracepcije po zdravljenju z zdravilom Zynteglo.
Hypokalaemia, Hypomagnesaemia
hiponatriemija, hipofosfatemija
Medicinal product subject to special and restricted medical prescription (see Annex I: Summary of Product Characteristics, section 4.2).
POGOJI ALI OMEJITVE GLEDE OSKRBE IN UPORABE Predpisovanje in izdaja zdravila je le na poseben recept in le s posebnim režimom (glejte Prilogo I: Povzetek glavnih značilnosti zdravila, poglavje 4.2).
The number
Številka serije/
The median (min, max) duration of follow-up was 40.48 (29.3, 58.6) months.
trajanje spremljanja je bilo 40,48 (29,3; 58,6) mesecev.
The median (min, max) total Hb at Month 6 for patients who had not received a transfusion for the prior 60 days was 11.90 (8.4, 13.3) g/dL (N=11).
skupnega Hb v 6. mesecu pri bolnikih, ki niso prejeli transfuzije v predhodnih 60 dneh, je znašala 11,90 (8,4; 13,3) g/dl (N=11).
(mL/kg/year) median (min, max)
(ml/kg/leto) mediana (min, maks)
• dizziness, tiredness
• omotica, utrujenost
The median (min, max) total Hb at Month 6 for patients who had not received a transfusion for the prior 60 days was 10.60 (7.6, 13.4) g/dL (N=11).
skupnega Hb v 6. mesecu pri bolnikih, ki niso prejeli transfuzije v predhodnih 60 dneh, je znašala 10,60 (7,6; 13,4) g/dL (N=11).
In clinical studies doses up to 20 × 106 CD34+ cells/kg have been administered.
V kliničnih študijah so uporabili odmerke do 20 × 106 CD34+ celic/kg.
Patients should not take anti-retroviral medicinal products or hydroxyurea from at least one month prior to mobilisation until at least 7 days after Zynteglo infusion (see section 4.4).
Bolniki ne smejo jemati protiretrovirusnih zdravil ali hidroksisečnine vsaj en mesec pred mobilizacijo ter do vsaj 7 dni po infuziji zdravila Zynteglo (glejte poglavje 4.4).
The median (min, max) time to last RBC transfusion was 0.46 (0.2, 5.8) months following Zynteglo infusion.
do zadnje transfuzije RBC je bila 0,46 (0,2; 5,8) mesecev po infuziji zdravila Zynteglo.
Zynteglo 1.2-20 × 106 cells/mL dispersion for infusion.
Zynteglo 1,2-20 × 106 celic/ml disperzija za infundiranje.
Insertion site analysis (ISA) of pre-transplantation transduced mouse BMCs and human CD34+ HSCs showed no enrichment for insertion in or near cancer-related genes.
Analiza mesta vstavitve (ISA) transduciranega CKM miši in humanih celic CD34+ HSC pred transplantacijo ni pokazala obogatitve za vstavljanje v gene,
Pre-medication for infusion reactions was managed at physician discretion.
Reakcije, povezane z infundiranjem zdravila Zynteglo Premediciranje za reakcije, povezane z infundiranjem, so obvladovali glede na presojo zdravnika.
Patients should not take anti-retroviral medications or hydroxyurea from at least one month prior to mobilisation until at least 7 days after Zynteglo infusion (see section 4.5).
Bolniki ne smejo jemati protiretrovirusnih zdravil ali hidroksisečnine vsaj en mesec pred mobilizacijo ter do vsaj 7 dni po infuziji zdravila Zynteglo (glejte poglavje 4.5).
Platelet counts should be monitored according to medical judgment until platelet engraftment and platelet recovery are achieved.
Po medicinski presoji je treba spremljati število trombocitov, dokler nista doseženi vsaditev in obnova trombocitov.
HbAT87Q remained generally stable through Month 24 with a median (min, max) of 6.44 (1.1, 10.1) g/dL (N=14, HGB-204 and HGB-205), and through long-term follow-up in LTF-303, demonstrating stable integration of the βA-T87Q-globin gene into long-term HSCs and stable expression of the βA-T87Q-globin gene in cells of the erythroid lineage.
6,44 (1,1; 10,1) g/dL (N=14, HGB-204 in HGB-205) in v času dolgoročnega spremljanja v LTF-303, pri čemer se je kazala stabilna integracija gena za βA-T87Q-globin v dolgoročnih celicah HSC in stabilno izražanje gena za βA-T87Q-globin v celicah eritroidne linije.
The effect of administration of Zynteglo to mothers on their breast-fed children has not been studied.
Učinek na dojene otroke pri dajanju zdravila Zynteglo doječim materam ni bil preučen.
Investigations
Študija
Depending on the myeloablative conditioning agent administered, prophylaxis for seizures should be considered (see section 5.1 for a description of the prophylaxis regimen used in clinical studies.)
Glede na učinkovino, uporabljeno za mieloablativni postopek, je treba razmisliti o profilaksi epileptičnih napadov (glejte poglavje 5.1 za opis režima profilakse, ki je bil uporabljen v kliničnih študijah).
Start of Zynteglo treatment
Začetek zdravljenja z zdravilom Zynteglo
• Physician educational material • Patient information pack
• izobraževalno gradivo za zdravnika
• bone or muscle pain
• bolečine v kosteh ali mišicah
Store in the vapour phase of liquid nitrogen at ≤-140°C until ready for thaw and administration.
Shranjujte v parni fazi tekočega dušika pri ≤ -140 °C do priprave za odtajanje in odmerjanje.
• feeling sick (nausea), being sick (vomiting)
• občutek slabosti (navzea), slabost (bruhanje)
Table 4
4. četrtletje
Among these 4 patients, the median (min, max) weighted average Hb during TI was 12.42 (11.5, 12.6) g/dL (Table 5).
obtežena povprečna vrednost Hb med TI znašala 12,42 (11,5; 12,6) g/dl (tabela 5).
o The need to explain and to ensure that patients understand: - potential risks of treatment with Zynteglo - signs of leukaemia/lymphoma and what action to take - content of patient's guide - the need to carry the patient alert card and show it to every healthcare professional - enrolment in the drug product Registry o Scope of the Registry and how to enrol patients
- znake levkemije/limfoma in kako naj ukrepa - vsebino vodnika za bolnika - potrebo, da ima opozorilno kartico za bolnika vedno s seboj in jo pokaže vsem zdravstvenim delavcem
Dispersion for infusion
Disperzija za infundiranje
• have abnormal bruising or bleeding (such as bruising without injury, blood in your urine, stool, vomit, or cough up blood).
• imate nenormalne modrice ali krvavitve (kot so modrice, ne da bi se poškodovali, kri v urinu,
The planned dose of busulfan was 0.8 mg/kg for patients 12-17 years of age as a 2-hour IV infusion every 6 hours for a total of 16 doses with a recommended target of AUC0-6h of 950-1125 µM*min.
Načrtovani odmerek busulfana je bil 3,2 mg/kg/dan za bolnike ≥ 18 let v obliki 3-urne intravenske infuzije dnevno v trajanju 4 dni s priporočeno ciljno AUC0-24 h v vrednosti 3800-4500 µM*min. Načrtovani odmerek busulfana je bil 0,8 mg/kg za bolnike, stare 12-17 let, v obliki 2-urne intravenske infuzije vsakih 6 ur do skupno 16 odmerkov s priporočeno ciljno AUC0-6 h 950-1125 µM*min. Za informacije o ustrezni metodi za določitev odmerka na podlagi telesne mase bolnika je bil uporabljen SmPC za busulfan.
• stomach pain, upset stomach, constipation, diarrhoea
• bolečine v trebuhu, razdražen želodec, zaprtost, driska
Zynteglo 1.2-20 × 106 cells/mL dispersion for infusion (autologous CD34+ cells encoding βA-T87Q-globin gene)
Zynteglo 1,2-20 × 106 celic/ml disperzija za infundiranje (avtologne celice CD34+, ki kodirajo gen za βA-T87Q-globin)
• skin abrasion/scrape
• kožne odrgnine/praske