TY - JOUR
T1 - Eltrombopag improves platelet engraftment after haploidentical bone marrow transplantation
T2 - Results of a Phase II study
AU - Ahmed, Sairah
AU - Bashir, Qaiser
AU - Bassett, Roland L.
AU - Ullah, Fauzia
AU - Aung, Fleur
AU - Valdez, Benigno C.
AU - Alousi, Amin M.
AU - Hosing, Chitra
AU - Kebriaei, Partow
AU - Khouri, Issa
AU - Marin, David
AU - Nieto, Yago
AU - Olson, Amanda
AU - Oran, Betul
AU - Qazilbash, Muzaffar H.
AU - Rezvani, Katayoun
AU - Mehta, Rohtesh
AU - Shpall, Elizabeth J.
AU - Ciurea, Stefan Octavian
AU - Andersson, Borje S.
AU - Champlin, Richard E.
AU - Popat, Uday R.
N1 - Publisher Copyright:
© 2024 Wiley Periodicals LLC.
PY - 2024/4
Y1 - 2024/4
N2 - Slow platelet recovery frequently occurs after haploidentical hematopoietic stem cell transplantation (haplo-HSCT) with bone marrow graft and post-transplant cyclophosphamide (PCy)-based graft-versus-host disease (GVHD) prophylaxis. Improved platelet recovery may reduce the need for transfusions and improve outcomes. We investigated the safety and efficacy of eltrombopag, a thrombopoietin receptor agonist, at enhancing platelet recovery post-haplo-HSCT. The prospective study included patients ≥18 years of age who received haplo-HSCT with bone marrow graft and PCy. Patients received eltrombopag 300 mg/day starting on Day +5. The primary objective was to estimate platelet engraftment (>50 000/μL by Day 60). In a post hoc analysis, they were compared to a contemporary matched control group who did not receive eltrombopag. One hundred ten patients were included in the analysis (30 eltrombopag and 80 control). Seventy-three percent and 50% of patients in the eltrombopag group and control group, respectively, attained >50 000/μL platelet count by Day 60 (p =.043). No eltrombopag-related grade ≥4 adverse events were observed. Median time to platelet recovery (>20 000/μL) was 29 days with eltrombopag and 31 days for controls (p =.022), while its cumulative incidence was 90% (95% confidence interval [CI]: 78%–100%) with eltrombopag versus 67.5% (95% CI: 57%–78%) for controls (p =.014). Number of platelet transfusions received, overall survival, progression-free survival, GVHD rate, relapse rate, and non-relapse mortality were similar between groups. Overall, eltrombopag is safe and improves platelet recovery in patients undergoing haplo-HSCT with bone marrow graft and PCy.
AB - Slow platelet recovery frequently occurs after haploidentical hematopoietic stem cell transplantation (haplo-HSCT) with bone marrow graft and post-transplant cyclophosphamide (PCy)-based graft-versus-host disease (GVHD) prophylaxis. Improved platelet recovery may reduce the need for transfusions and improve outcomes. We investigated the safety and efficacy of eltrombopag, a thrombopoietin receptor agonist, at enhancing platelet recovery post-haplo-HSCT. The prospective study included patients ≥18 years of age who received haplo-HSCT with bone marrow graft and PCy. Patients received eltrombopag 300 mg/day starting on Day +5. The primary objective was to estimate platelet engraftment (>50 000/μL by Day 60). In a post hoc analysis, they were compared to a contemporary matched control group who did not receive eltrombopag. One hundred ten patients were included in the analysis (30 eltrombopag and 80 control). Seventy-three percent and 50% of patients in the eltrombopag group and control group, respectively, attained >50 000/μL platelet count by Day 60 (p =.043). No eltrombopag-related grade ≥4 adverse events were observed. Median time to platelet recovery (>20 000/μL) was 29 days with eltrombopag and 31 days for controls (p =.022), while its cumulative incidence was 90% (95% confidence interval [CI]: 78%–100%) with eltrombopag versus 67.5% (95% CI: 57%–78%) for controls (p =.014). Number of platelet transfusions received, overall survival, progression-free survival, GVHD rate, relapse rate, and non-relapse mortality were similar between groups. Overall, eltrombopag is safe and improves platelet recovery in patients undergoing haplo-HSCT with bone marrow graft and PCy.
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U2 - 10.1002/ajh.27233
DO - 10.1002/ajh.27233
M3 - Article
C2 - 38314663
AN - SCOPUS:85184214720
SN - 0361-8609
VL - 99
SP - 562
EP - 569
JO - American journal of hematology
JF - American journal of hematology
IS - 4
ER -