2019
DOI: 10.1097/mph.0000000000001263
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Eltrombopag for Delayed Platelet Recovery and Secondary Thrombocytopenia Following Allogeneic Stem Cell Transplantation in Children

Abstract: The delay in platelet recovery after hematopoietic stem cell transplantation (HSCT) is closely related to the overall survival rate of transplanted children. The use of platelet-producing agents such as eltrombopag and romiplostim has made great progress in treating diseases such as immune thrombocytopenia and aplastic anemia. However, the use of such drugs in patients with thrombocytopenia after transplantation, especially in children, is rare. This study aimed to report eltrombopag treatment for 3 children w… Show more

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Cited by 17 publications
(14 citation statements)
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“…Thus, eltrombopag seems a good candidate agent to be investigated in the setting of post-HSCT thrombocytopenia or PoGF, which are obviously associated with a similar quantitative impairment of the HSC compartment [19]. Some few anecdotic cases have been described in the last 2 years; [24][25][26][27][28][29][30][31][32][33] however, data from clinical trials are not available, with the exception of a preliminary report from a phase II placebo-controlled randomized trial [34]. In this study, which enrolled 60 transplanted (53 allogeneic and 7 autologous) patients with persistent thrombocytopenia or neutropenia, the response rate in the experimental arm was 36%, but results were statistically inconclusive in terms of superiority in comparison with the control arm (28% responses) [34].…”
Section: Discussionmentioning
confidence: 99%
“…Thus, eltrombopag seems a good candidate agent to be investigated in the setting of post-HSCT thrombocytopenia or PoGF, which are obviously associated with a similar quantitative impairment of the HSC compartment [19]. Some few anecdotic cases have been described in the last 2 years; [24][25][26][27][28][29][30][31][32][33] however, data from clinical trials are not available, with the exception of a preliminary report from a phase II placebo-controlled randomized trial [34]. In this study, which enrolled 60 transplanted (53 allogeneic and 7 autologous) patients with persistent thrombocytopenia or neutropenia, the response rate in the experimental arm was 36%, but results were statistically inconclusive in terms of superiority in comparison with the control arm (28% responses) [34].…”
Section: Discussionmentioning
confidence: 99%
“…[16][17][18][19][20] In children in the same context, data are limited to scarce case reports. [21][22][23] To our knowledge, there have been no studies on the use of eltrombopag to treat PGF post-HSCT in children. Our retrospective series, though small, shows that eltrombopag was both effective and safe in this setting, with results similar to those reported in adults.…”
Section: Discussionmentioning
confidence: 99%
“…Most studies have analyzed its use in the treatment of post‐HSCT thrombocytopenia in adults and have reported response rates between 57% and 72% and a favorable toxicity profile 16‐20 . In children in the same context, data are limited to scarce case reports 21‐23 …”
Section: Discussionmentioning
confidence: 99%
“…There is less experience in children. [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.…”
Section: Posttransplantmentioning
confidence: 99%