Mayu 2023: Hukumar Abinci da Magunguna ta amince da Omidubicel-onlv (Omisirge, Gamida Cell Ltd.) don amfani da manya da marasa lafiya na yara (shekaru 12 da haihuwa) tare da cututtukan cututtukan jini waɗanda aka tsara don karɓar dashen jini na cibiya bayan yanayin yanayin myeloablative. don hanzarta dawo da neutrophil kuma rage haɗarin kamuwa da cuta.
A cikin Nazarin P0501 (NCT02730299), alamar buɗewa, multicenter, gwajin bazuwar dasawa na omidubicel-onlv ko juzu'in dashen igiyar igiya (UCB) bayan yanayin yanayin myeloablative a cikin marasa lafiya da cututtukan hematologic, an kimanta tasiri da amincin jiyya. An ba da jimillar mutane 125 ba da gangan ba, tare da 62 sun karɓi omidubicel-onlv da 63 sun karɓi UCB. Marasa lafiya 52 sun sami dashen omidubicel-onlv, tare da matsakaicin matsakaici na 9.0 X 106 sel / kg (kewayon 2.1 - 47.6 X 106 sel / kg) na sel CD34+. A cikin hannun UCB, marasa lafiya 56 suna da raka'a ɗaya ko biyu (66% sun karɓi raka'a igiya biyu) dasa. Matsakaicin adadin CD34+ na tsakiya a cikin marasa lafiya na 42 waɗanda ke da allurai na sel bayan narke da aka rubuta shine 0.2 X 106 sel / kg (kewayon 0.0 - 0.8 X 106 sel / kg). Akwai wasu ƙa'idodin kwantar da hankali da aka yi amfani da su, kamar waɗanda suka dogara akan chemotherapy ko Jimillar Irradiation na Jiki.
Lokacin da za a dawo da neutrophil bayan dasawa da kuma yawan Jini da Marrow Transplant Clinical Trials Network (BMT CTN) Grade 2/3 kwayoyin cuta ko cututtukan fungal na Grade 3 ta hanyar Rana ta 100 bayan dasawa sune matakan sakamako na farko. Matsakaicin lokacin dawowar neutrophil shine kwanaki 12 (95% CI: 10-15 days) ga waɗanda ke karɓar omidubicel-onlv, da kwanaki 22 (95% CI: 19-25 days) ga waɗanda ke karɓar UCB. A cikin hannun omidubicel-onlv, 87% na marasa lafiya da 83% na waɗanda ke karɓar UCB sun sami farfadowar neutrophil. Ta hanyar Rana ta 100 bayan dasawa, abin da ya faru na BMT CTN Grade 2/3 kwayoyin cuta ko cututtukan fungal na Grade 3 shine 39% da 60%, bi da bi, a cikin ƙungiyoyin biyu.
Abubuwan da aka ba da izini sun haɗa da Gargaɗi na Akwati don halayen jiko mai mutuƙar mutuwa ko mai barazanar rai, cutarwa da cututtukan gida (GvHD), ciwo na engraftment, da gazawar sawa, kama da na magungunan UCB masu izini. An gudanar da Omidubicel-onlv ga mutane 117 don kowace cuta; daga cikinsu, 47% sun sami halayen jiko, 58% sun sami GVHD mai tsanani, 35% sun sami GVHD na yau da kullun, da kuma 3% sun sami gazawar graft.
Mafi yawan martani mara kyau na Grade 3-5 a cikin Nazarin P0501 marasa lafiya tare da cututtukan hematologic sun kasance zafi (33%), kumburin mucosal (31%), hauhawar jini (25%), da guba na gastrointestinal (19%).
Shawarar da aka ba da shawarar omidubicel-onlv shine jiko na jere guda biyu wanda ya ƙunshi masu zuwa:
- Ƙarshen Al'ada: mafi ƙarancin 8.0 × 108 jimillar sel masu iya aiki tare da mafi ƙarancin 8.7 bisa dari CD34+ da ƙaramar 9.2 × 107 jimlar sel CD34+, sannan
- juzu'in da ba a al'ada ba: mafi ƙarancin 4.0 × 108 jimlar sel masu aiki tare da mafi ƙarancin 2.4 × 107 CD3+ Kwayoyin.
