TY - JOUR
T1 - Eltrombopag for Post-Transplantation Thrombocytopenia
T2 - Results of Phase II Randomized, Double-Blind, Placebo-Controlled Trial: S. Ahmed et al
AU - Ahmed, Sairah
AU - Bashir, Qaiser
AU - Bassett, Roland
AU - Poon, Man Yin C.
AU - Valdez, Ben
AU - Konoplev, Sergej
AU - Alousi, Amin M.
AU - Andersson, Borje S.
AU - Ciurea, Stefan
AU - Hosing, Chitra
AU - Jones, Roy
AU - Kebriaei, Partow
AU - Khouri, Issa
AU - Kim, Stella
AU - Nieto, Yago
AU - Olson, Amanda
AU - Oran, Betul
AU - Parmar, Simrit
AU - Qazilbash, Muzaffar H.
AU - Rezvani, Katyoun
AU - Shah, Nina
AU - Shpall, E. J.
AU - Champlin, Richard
AU - Popat, Uday
N1 - Publisher Copyright:
© 2021 The American Society for Transplantation and Cellular Therapy
PY - 2021/5
Y1 - 2021/5
N2 - Prolonged thrombocytopenia occurs in up to 37% of patients after hematopoietic stem cell transplantation (HSCT) and is associated with adverse prognosis and increased risk of bleeding. Eltrombopag, a thrombopoietin receptor agonist, can increase platelet counts in thrombocytopenic patients. We conducted a phase II study, adaptively randomizing patients at ≥35 days post-HSCT to receive placebo or eltrombopag at a platelet count ≤20,000/µL for 7 days or platelet transfusion-dependent and a neutrophil count ≥1500/µL. Sixty patients were randomized to eltrombopag (n = 42) or placebo (n = 18) and received at least 1 dose. Fifteen patients (36%) in the eltrombopag arm achieved a platelet count of ≥30,000/µL, compared with 5 patients (28%) in the placebo arm, with a posterior probability of 0.75. (The protocol required this probability to be >0.975 to declare a winner; thus, the results are inconclusive.) However, 9 patients (21%) in the eltrombopag arm achieved a platelet count of ≥50,000/µL, compared with no patients in the placebo arm (P = .046). The overall survival, progression-free survival, relapse rate, and nonrelapse mortality were similar in the 2 arms. In conclusion, compared with placebo, treatment with eltrombopag led to a higher percentage of patients achieving a platelet count of ≥50,000/µL in patients with persistent thrombocytopenia after HSCT.
AB - Prolonged thrombocytopenia occurs in up to 37% of patients after hematopoietic stem cell transplantation (HSCT) and is associated with adverse prognosis and increased risk of bleeding. Eltrombopag, a thrombopoietin receptor agonist, can increase platelet counts in thrombocytopenic patients. We conducted a phase II study, adaptively randomizing patients at ≥35 days post-HSCT to receive placebo or eltrombopag at a platelet count ≤20,000/µL for 7 days or platelet transfusion-dependent and a neutrophil count ≥1500/µL. Sixty patients were randomized to eltrombopag (n = 42) or placebo (n = 18) and received at least 1 dose. Fifteen patients (36%) in the eltrombopag arm achieved a platelet count of ≥30,000/µL, compared with 5 patients (28%) in the placebo arm, with a posterior probability of 0.75. (The protocol required this probability to be >0.975 to declare a winner; thus, the results are inconclusive.) However, 9 patients (21%) in the eltrombopag arm achieved a platelet count of ≥50,000/µL, compared with no patients in the placebo arm (P = .046). The overall survival, progression-free survival, relapse rate, and nonrelapse mortality were similar in the 2 arms. In conclusion, compared with placebo, treatment with eltrombopag led to a higher percentage of patients achieving a platelet count of ≥50,000/µL in patients with persistent thrombocytopenia after HSCT.
KW - Eltrombopag
KW - Hematopoietic stem cell transplantation
KW - Thrombocytopenia
UR - http://www.scopus.com/inward/record.url?scp=85102107976&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85102107976&partnerID=8YFLogxK
U2 - 10.1016/j.jtct.2021.02.004
DO - 10.1016/j.jtct.2021.02.004
M3 - Article
C2 - 33965187
AN - SCOPUS:85102107976
SN - 2666-6367
VL - 27
SP - 430.e1-430.e7
JO - Transplantation and Cellular Therapy
JF - Transplantation and Cellular Therapy
IS - 5
ER -