Eltrombopag for thrombocytopenia following allogeneic hematopoietic stem cell transplantation in children

Abstract

Persistent thrombocytopenia is a common complication after allogeneic hematopoietic stem cell transplantation (HSCT). While the use of thrombopoietin receptor agonists was retrospectively investigated in adults, data in pediatric posttransplant thrombocytopenia are lacking. We evaluated the safety and efficacy of eltrombopag in nine children with platelet transfusion‐dependent persistent thrombocytopenia after HSCT. Eltrombopag was started at a median of 147 days after allo‐SCT and continued for a median period of 64 days, the starting dose being 50 mg per day. The therapy was well tolerated. After a median time of treatment of 36 days, eight patients (88%) reached sustained platelets count >50 000/μL.

Eltrombopag for treatment of thrombocytopenia after allogeneic hematopoietic cell transplantation in children: Single‐centre experience

“…In none of these childhood studies and case reports, the maximum daily dose of ELT did not exceed 75 mg as used in ITP. 8,13,15,17 But in our study, we used a higher dosage reaching 150 mg/d, for 4 of our patients monitoring the platelet response, as used in AA. Although liver injury has been reported in some studies with a frequency of 0%-13.2%, none of the patients in these studies discontinue ELT because of adverse events or intolerability.…”

“…Several studies have reported the efficiency of romiplostim and ELT for the treatment of thrombocytopenia after alloHSCT in adults, but there are only some case reports and one retrospective study with ELT treatment after pediatric alloHSCT. [8][9][10][11][12][13][14][15][16][17] This is the second and largest retrospective study about the efficacy of ELT use after alloHSCT in childhood. 8 alloHSCT.…”

“…[8][9][10][11][12][13][14][15][16][17] This is the second and largest retrospective study about the efficacy of ELT use after alloHSCT in childhood. 8 alloHSCT. SFPR was defined as a decline of platelet counts below 30 000/µL lasting at least 7 consecutive days or requiring platelet transfusions within 7 days after achieving primary platelet recovery.…”

A Review of Romiplostim Mechanism of Action and Clinical Applicability

“…[171][172][173][174][175] In particular, an Italian retrospective study recently reported on the use of eltrombopag in nine pediatric patients after HSCT; after a median treatment time of 36 days, eight of the nine patients (88%) achieved sustained platelet counts >50 × 10 9 /L. 173 In seven children with secondary failure of platelet recovery treated with romiplostim, six (86%) became transfusion-independent in the second week of treatment. 174 These studies suggest romiplostim could be safely administered to patients with transfusion-dependent thrombocytopenia after allogeneic HSCT and may improve platelet counts.…”

Comment on: Eltrombopag for thrombocytopenia following allogeneic hematopoietic stem cell transplantation in children

“…They have demonstrated that nine platelet-transfusion-dependent patients became independent from transfusions with a median interval between starting ELT to the last transfusion of 14 days, and 88% of the patients reached sustained platelet counts >50 000/ L after a median time of 36 days from starting ELT. 1 Recently, we have also investigated ELT responses in three children Failure of platelet recovery due to poor graft function, relapse of primary disease, viral infections, drug toxicity, and immune processes can occur after HSCT. 2 Treatment options are limited for thrombocytopenia caused by poor platelet production.…”