New England Journal of Medicine· 2018Q1
B Hücreli Lenfoblastik Lösemide Tisagenlecleucel'in Çocuklarda ve Genç Yetişkinlerde Kullanımı
Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia
- 5.682atıf
- Q1SCImago
- 2018yıl
Kısa özet
Tek doz anti-CD19 CAR T-hücre tedavisi olan tisagenlecleucel, tekrarlayan veya refrakter B hücreli ALL'li pediatrik ve genç hasta popülasyonunda 3 ay içinde %81'lik genel remisyon oranı sağlamış ve 12 aya kadar kalıcı remisyonlar gözlemlenmiştir.
Yapay zekâ ile başlık ve abstract'tan üretildi; tam metin okunmaz.
Ana noktalar
- Tisagenlecleucel, tekrarlayan/refrakter B hücreli ALL'li pediatrik/genç hastalarda 3 ay içinde %81'lik genel remisyon oranı elde etmiştir.
- Remisyona ulaşan tüm hastalarda minimal artık hastalık saptanmamıştır.
- 6 aylık olaylardan bağımsız sağkalım %73 ve genel sağkalım %90 olmuştur.
- Hastaların %73'ünde sitokin salınım sendromu (%77) ve nörolojik olaylar (%40) dahil olmak üzere derece 3 veya 4 yan etkiler görülmüştür.
- Tisagenlecleucel'in kan dolaşımında 20 aya kadar gözlemlenmesi, uzun süreli kalıcılık olduğunu düşündürmektedir.
Yapay zekâ ile başlık ve abstract'tan üretildi; tam metin okunmaz.
Özet (abstract)
BACKGROUND: In a single-center phase 1-2a study, the anti-CD19 chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel produced high rates of complete remission and was associated with serious but mainly reversible toxic effects in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). METHODS: We conducted a phase 2, single-cohort, 25-center, global study of tisagenlecleucel in pediatric and young adult patients with CD19+ relapsed or refractory B-cell ALL. The primary end point was the overall remission rate (the rate of complete remission or complete remission with incomplete hematologic recovery) within 3 months. RESULTS: For this planned analysis, 75 patients received an infusion of tisagenlecleucel and could be evaluated for efficacy. The overall remission rate within 3 months was 81%, with all patients who had a response to treatment found to be negative for minimal residual disease, as assessed by means of flow cytometry. The rates of event-free survival and overall survival were 73% (95% confidence interval [CI], 60 to 82) and 90% (95% CI, 81 to 95), respectively, at 6 months and 50% (95% CI, 35 to 64) and 76% (95% CI, 63 to 86) at 12 months. The median duration of remission was not reached. Persistence of tisagenlecleucel in the blood was observed for as long as 20 months. Grade 3 or 4 adverse events that were suspected to be related to tisagenlecleucel occurred in 73% of patients. The cytokine release syndrome occurred in 77% of patients, 48% of whom received tocilizumab. Neurologic events occurred in 40% of patients and were managed with supportive care, and no cerebral edema was reported. CONCLUSIONS: In this global study of CAR T-cell therapy, a single infusion of tisagenlecleucel provided durable remission with long-term persistence in pediatric and young adult patients with relapsed or refractory B-cell ALL, with transient high-grade toxic effects. (Funded by Novartis Pharmaceuticals; ClinicalTrials.gov number, NCT02435849 .).
Yazarların özeti; kaynağından alınmıştır. New England Journal of Medicine, 2018 · DOI ↗
Ücretsiz hesapla devam et
Makaleye Sor ile bu makaleye günde 3 soru ücretsiz; makaleyi kaydet, kaynakçasını al, ilgi alanına göre her gün yeni özetler. Çıkarımlar Premium.
Web'de ücretsiz devam etGoogle ya da Apple hesabınla giriş; kart istemez. Bu makaleye geri dönersin.
Telefonda:
Alan: Onkoloji
OncologyMedicine