Blinatumomab in Children and Adolescents with Relapsed/Refractory B Cell Precursor Acute Lymphoblastic Leukemia: A Real-Life Multicenter Retrospective Study in Seven AIEOP (Associazione Italiana di Ematologia e Oncologia Pediatrica) Centers

Cancers

Pio Stellato¹  Giuseppe Menna¹  Rosanna Parasole¹  Antonia De Matteo¹  Fara Petruzziello¹  Giuliana Beneduce¹  Maria C. Putti²  Anna M. Testi³  Simone Cesaro⁴  Monica Cellini⁵  Antonella Colombini⁶  Carmelo Rizzari⁶  Elena Barisone⁷  Nicoletta Bertorello⁷ 

[1] Department of Pediatric Hemato-Oncology, AORN Santobono-Pausilipon, Hospital, 80123 Naples, Italy;Department of Woman and Child Health, Clinic of Pediatric Haematology-Oncology, University of Padova, 35128 Padova, Italy;Division of Hematology, Department of Translational and Precision Medicine, ‘Sapienza’ University, 00161 Rome, Italy;Pediatric Hematology Oncology Unit, Department of Mother and Child, Azienda Ospedaliera Universitaria Integrata, 37129 Verona, Italy;Pediatric Hematology Oncology Unit, Department of Mother and Child, Azienda Ospedaliero Universitaria Policlinico di Modena, 41125 Modena, Italy;Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, MBBM Foundation c/o ASST Monza, 20900 Milan, Italy;Stem Cell Transplantation and Cell Therapy Division, Department of Pediatric Onco-Hematology, A.O.U. Città della Salute e della Scienza—Regina Margherita Children’s Hospital, 10126 Turin, Italy;

关键词: targeted therapy;    acute lymphoblastic leukemia;    pediatric;    relapse;    toxicity;    real-life experience;   

DOI  :  10.3390/cancers14020426

来源: DOAJ

【 摘 要 】

Five-year event-free survival in pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL) currently exceeds 80–85%. However, 15–20% of patients still experience a relapsed/refractory disease. From 1 January 2015 to 31 December 2020, thirty-nine patients, 0–21 years old with r/r BCP-ALL were treated with blinatumomab with the aim of inducing remission (n = 13) or reducing MRD levels (n = 26) in the frame of different multiagent chemotherapy schedules, in seven AIEOP centers. Patients were treated in compassionate and/or off-label settings and were not enrolled in any controlled clinical trials. Treatment was well tolerated; 22 (56.4%) patients reported adverse events (AE) on a total of 46 events registered, of which 27 (58.7%) were ≤2 grade according to CTCAE. Neurological AEs were 18 (39.1%); only two patients required transient blinatumomab discontinuation. Complete remission (CR) rate was 46% for the 13 patients treated with ≥5% blasts and 81% PCR/FC MRD negativity in the 26 patients with blasts < 5%. Median relapse-free survival was 33.4 months (95% CI; 7.5–59.3); median overall survival was not reached over a mean follow-up of 16 months. In our study, as in other real-life experiences, blinatumomab proved to be effective and well-tolerated, able to induce a high rate of CR and MRD negativity.

【 授权许可】

Unknown