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Vol. 42. Issue S1.
Pages 31 (October 2020)
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Vol. 42. Issue S1.
Pages 31 (October 2020)
OP 26
Open Access
Eltrombopag for thrombocytopenia following pediatric allogeneic hematopoietic stem cell transplantation
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A. Akyay*, Y. Oncul
Inonu University School of Medicine, Malatya, Turkey
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Objective: Failure of platelet recovery is a complication occurring after allogeneic hematopoietic stem cell transplantation (HSCT). Poor graft function, relapse, viral infections, drug toxicity, immune processes may lead to decreased platelets production. Treatment options are limited for thrombocytopenia caused by poor platelet production. While the use of Eltrombopag (ELT) was retrospectively investigated in adult patients, data regarding the potential benefit of these agent for pediatric posttransplant thrombocytopenia are lacking. We report three pediatric patients who received ELT for thrombocytopenia occurring after HSCT.

Case report: The median patients’ age at HSCT was 13.3 years (10–18). All patients had HSCT from a sibling donor with the bone marrow stem cell source. All patients were treated with a myeloablative conditioning regimen. Patients engrafted at a median time of 19 days (10–24) for neutrophils and 49 days (44–49) for platelets. Bone marrow aspirates showed a decrease number of megakaryocytes, and all patients had been ineffectively treated with high-dose intravenous gamma globulin and with steroids before ELT initiation.

Methodology: ELT was started at a median time after HSCT of 57 days (42–90), the starting dose being 25mg/day, and the maximum administered dose was 75mg/day. ELT was continued for a median period of 64 days (28–286). All patients reached sustained platelets count >50,000/μL after a median time from starting ELT of 197 days (87–210). The median platelet count at last evaluation was 115,000/μL (range 66,000–125,000/μL). ELT was well tolerated, and no patient have developed important side effect.

Results: Our cases became transfusion independent after a median time from starting ELT of 197 days. In the pediatric post-HSCT setting, only few previously published case reports described the successful use of ELT as a treatment for thrombocytopenia. Li et al. reported three children transplanted for nonmalignant disease treated for both primary and secondary failure of platelet engraftment. Treatment was effective in two patients, but not in one patient transplanted for Gaucher disease. In Masettis’ study, the 60-day cumulative incidence of platelet recovery >50,000/μL after ELT treatment was 75%. Similarly, Tanaka et al. described 12 adults treated for primary and secondary post-HSCT thrombocytopenia who reached platelet count>50,000/μL in 60% and 71% of cases respectively.

Conclusion: Our study supports the safety and efficacy of ELT for treatment of prolonged thrombocytopenia after allogeneic HSCT in children. Future prospective studies are needed to confirm these findings.

Idiomas
Hematology, Transfusion and Cell Therapy
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