Eltrombopag for Post-Transplantation Thrombocytopenia: Results of Phase II Randomized, Double-Blind, Placebo-Controlled Trial

Ahmed, Sairah; Bashir, Qaiser; Bassett, Roland L.; Poon, Man-Yin C.; Valdez, Ben C.; Konoplev, Sergej; Alousi, Amin M.; Andersson, Börje S.; Ciurea, Stefan O.; Hosing, Chitra; Jones, R. D.; Kebriaei, Partow; Khouri, Issa F.; Kim, Stella; Nieto, Yago; Olson, Amanda; Oran, Betül; Parmar, Simrit; Qazilbash, Muzaffar H.; Rezvani, Katyoun; Shah, Nina; Shpall, E.J.; Champlin, Richard E.; Popat, Uday

doi:10.1016/j.jtct.2021.02.004

Cited by 20 publications

(18 citation statements)

References 19 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…In a phase II randomized trial of 60 patients with prolonged post-HCT thrombocytopenia, a significantly higher proportion of patients in the eltrombopag arm achieved a platelet count of $50,000/mcL compared with the placebo arm (21% vs 0%; P5.046). 16 However, OS, PFS, relapse rate, and nonrelapse mortality were similar in the 2 arms. These data suggest that TPO-RAs can promote platelet recovery and reduce platelet transfusion in patients with prolonged post-HCT thrombocytopenia, but more robust data are needed.…”

Section: Prolonged Posthematopoietic Cell Transplant Thrombocytopeniamentioning

confidence: 90%

“…14 Several studies have examined the efficacy of TPO-RAs in patients with prolonged thrombocytopenia following HCT, including those with secondary failure of platelet recovery. [14][15][16] An extensive systematic review of 37 case reports suggests that patients with prolonged post-HSCT thrombocytopenia may respond to either eltrombopag (overall response rate [ORR], 70%) or romiplostim (ORR, 82%), with no evidence of serious adverse effects. However, the strength of the evidence is weak due to the retrospective nature of the studies, lack of control groups, and heterogeneity of the data.…”

Section: Prolonged Posthematopoietic Cell Transplant Thrombocytopeniamentioning

confidence: 99%

See 1 more Smart Citation

NCCN Guidelines® Insights: Hematopoietic Growth Factors, Version 1.2022

Griffiths¹,

Roy²,

Alwan

et al. 2022

Journal of the National Comprehensive Cancer Network

View full text Add to dashboard Cite

The NCCN Guidelines for Hematopoietic Growth Factors provide recommendations for the appropriate use of growth factors in the clinical management of febrile neutropenia (FN), chemotherapy-induced thrombocytopenia (CIT), and chemotherapy-induced anemia (CIA). Management and prevention of these sequelae are an integral part of supportive care for many patients undergoing cancer treatment. The purpose of these guidelines is to operationalize the evaluation, prevention, and treatment of FN, CIT, and CIA in adult patients with nonmyeloid malignancies and to enable the patient and clinician to assess management options for FN, CIT, and CIA in the context of an individual patient’s condition. These NCCN Guidelines Insights provide a summary of the important recent updates to the NCCN Guidelines for Hematopoietic Growth Factors, with particular emphasis on the incorporation of a newly developed section on CIT.

show abstract

Section: Prolonged Posthematopoietic Cell Transplant Thrombocytopeniamentioning

confidence: 90%

Section: Prolonged Posthematopoietic Cell Transplant Thrombocytopeniamentioning

confidence: 99%

NCCN Guidelines® Insights: Hematopoietic Growth Factors, Version 1.2022

Griffiths¹,

Roy²,

Alwan

et al. 2022

Journal of the National Comprehensive Cancer Network

View full text Add to dashboard Cite

show abstract

“…al. 40 There are some limitations in this trial. Due to small number of participants enrolled, it was difficult to analyzed in the subgroup, and the results may be biased.…”

Section: Discussionmentioning

confidence: 96%

“…This study found that no statistical difference in the rate of a‐GVHD or virus‐related infections between the two groups, which is consistent with the results of Ahmed et. al 40 …”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Oral eltrombopag versus subcutaneous recombinant human thrombopoietin for promoting platelet engraftment after allogeneic stem cell transplantation: A prospective, non‐inferiority, randomized controlled trial

Wen

Zhang

Chen

et al. 2022

Hematological Oncology

View full text Add to dashboard Cite

Delayed platelet engraftment (DPE) is associated with poor survival and increased transplantation‐related mortality after allogeneic hematopoietic stem cell transplantation (allo‐HSCT). Therefore, treatments are needed to improve platelet engraftment and prevent DPE. We performed a phase three, non‐inferior, randomized controlled study of eltrombopag or recombinant human thrombopoietin (rhTPO) to promot platelet engraftment after allo‐HSCT. Candidates for allo‐HSCT were randomly assigned to receive oral eltrombopag (50 mg daily) or subcutaneous rhTPO (15000U daily) from the first‐day post‐transplantation. The primary endpoint was the cumulative numbers of platelet engraftment (platelet recovery ≥20 × 109/L, without transfusion, for seven consecutive days) on day 60 after transplantation. We performed intention‐to‐treat analyses with a non‐inferior margin of −15%. A total of 92 participants underwent randomization. 44 and 48 patients were randomized to the eltrombopag and rhTPO groups, respectively. The median duration of follow‐up was 360 days (range: 12–960 days). The cumulative incidence of platelet engraftment on day 60 after transplantation in eltrombopag group was 86.4% (38/44) compared with 85.4% (41/48) in the rhTPO group (absolute risk difference [ARD] 1%, one‐sided lower limit of 95% confidence interval [CI] −13.28%, Pnon‐inferirioty = 0.014). The rate of DPE in the eltrombopag group was 6.8% (3/44) compared with 12.5% (6/48) in the rhTPO group (ARD ‐5.7%, one‐sided higher limit of 95% CI 6.28%, Pnon‐inferirioty = 0.063). Approximately, three‐fourths of non‐hematologic adverse events were not observed in the eltrombopag group but three patients (3/48, 6%) experienecd them in the rhTPO group. In addition, platelet transfusions unite from day 0 to day 21, or from day 22 to day 60, progression‐free survival, overall survival were not significantly different between both groups. Eltrombopag was non‐inferior to rhTPO in promoting platelet engraftment post allo‐HSCT for patients with hematological malignancy. Oral eltrombopag was more convenient for patients than subcutaneous rhTPO (NCT03515096).

show abstract

Alternatives, and adjuncts, to prophylactic platelet transfusion for people with haematological malignancies undergoing intensive chemotherapy or stem cell transplantation

Desborough

Estcourt

Dorée

et al. 2016

Cochrane Database of Systematic Reviews

View full text Add to dashboard Cite

There is insufficient evidence to determine if platelet-poor plasma or TPO mimetics reduce bleeding for participants with haematological malignancies undergoing intensive chemotherapy or stem cell transplantation. To detect a decrease in the proportion of participants with clinically significant bleeding from 12 in 100 to 6 in 100 would require a trial containing at least 708 participants (80% power, 5% significance). The six ongoing trials will provide additional information about the TPO mimetic comparison (424 participants) but this will still be underpowered to demonstrate this level of reduction in bleeding. None of the included or ongoing trials include children. There are no completed or ongoing trials assessing artificial platelet substitutes, fibrinogen concentrate, recombinant activated factor VII or desmopressin in people undergoing intensive chemotherapy or stem cell transplantation for haematological malignancies.

show abstract

Eltrombopag for Post-Transplantation Thrombocytopenia: Results of Phase II Randomized, Double-Blind, Placebo-Controlled Trial

Cited by 20 publications

References 19 publications

NCCN Guidelines® Insights: Hematopoietic Growth Factors, Version 1.2022

NCCN Guidelines® Insights: Hematopoietic Growth Factors, Version 1.2022

Oral eltrombopag versus subcutaneous recombinant human thrombopoietin for promoting platelet engraftment after allogeneic stem cell transplantation: A prospective, non‐inferiority, randomized controlled trial

Alternatives, and adjuncts, to prophylactic platelet transfusion for people with haematological malignancies undergoing intensive chemotherapy or stem cell transplantation

Contact Info

Product

Resources

About