scholarly journals Efficacy and Safety of Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) for 67 Pediatric Patients with High Risk T-Cell Acute Lymphoblastic Leukemia (T-ALL)

2019 ◽  
Vol 25 (3) ◽  
pp. S111-S112
Author(s):  
Rui-Juan Sun ◽  
Zhi-Jie Wei ◽  
De-Yan Liu ◽  
Xing-Yu Cao ◽  
Jian-Ping Zhang ◽  
...  
Keyword(s):  
Stem Cell ◽  
Acute Lymphoblastic Leukemia ◽  
High Risk ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Pediatric Patients ◽  
Lymphoblastic Leukemia ◽  
Efficacy And Safety ◽  
Hematopoietic Stem ◽  
Cell Acute Lymphoblastic Leukemia

scholarly journals Phase 2 trial of clofarabine in combination with etoposide and cyclophosphamide in pediatric patients with refractory or relapsed acute lymphoblastic leukemia

Blood ◽  
2011 ◽  
Vol 118 (23) ◽  
pp. 6043-6049 ◽  
Author(s):  
Nobuko Hijiya ◽  
Blythe Thomson ◽  
Michael S. Isakoff ◽  
Lewis B. Silverman ◽  
Peter G. Steinherz ◽  
...  
Keyword(s):  
Stem Cell ◽  
Acute Lymphoblastic Leukemia ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Pediatric Patients ◽  
Overall Response Rate ◽  
Response Rate ◽  
Lymphoblastic Leukemia ◽  
Phase 2 ◽  
Hematopoietic Stem

Abstract The outcomes in children with refractory/relapsed (R/R) acute lymphoblastic leukemia (ALL) are dismal. The efficacy and safety of intravenous clofarabine 40 mg/m2 per day, cyclophosphamide 440 mg/m2 per day, and etoposide 100 mg/m2 per day for 5 consecutive days in pediatric patients with R/R ALL was evaluated in this phase 2 study. The primary endpoint was overall response rate (complete remission [CR] plus CR without platelet recovery [CRp]). Among the 25 patients (median age, 14 years; pre-B cell ALL, 84%; ≥ 2 prior regimens: 84%; refractory to previous regimen: 60%), the overall response rate was 44% (7 CR, 4 CRp) with a 67.3-week median duration or remission censored at last follow-up. Most patients proceeded to alternative therapy, and 10 patients (40%) received hematopoietic stem cell transplantation. Six patients (24%) died because of treatment-related adverse events associated with infection, hepatotoxicity, and/or multiorgan failure. The study protocol was amended to exclude patients with prior hematopoietic stem cell transplantation after 4 of the first 8 patients developed severe hepatotoxicity suggestive of veno-occlusive disease. No additional cases of veno-occlusive disease occurred. The regimen offered encouraging response rates and sustained remission in R/R patients. Future investigation should include exploration of patient selection, dosing, and supportive care. This trial was registered at www.clinicaltrials.gov as #NCT00315705.

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