Avatrombopag for severe refractory thrombocytopenia in a pediatric patient with ALL following allogeneic hematopoietic stem cell transplantation: A case report.

Autor:	Lynch EJ; Division of Pediatric Hematology, Oncology, Stem Cell Transplantation, University of Florida, Shands Children's Hospital, Gainesville, FL, USA., Citta A; Division of Pediatric Hematology, Oncology, Stem Cell Transplantation, University of Florida, Shands Children's Hospital, Gainesville, FL, USA., Alford C; Division of Pediatric Hematology, Oncology, Stem Cell Transplantation, University of Florida, Shands Children's Hospital, Gainesville, FL, USA., Ligon JA; Division of Pediatric Hematology, Oncology, Stem Cell Transplantation, University of Florida, Shands Children's Hospital, Gainesville, FL, USA., Dalal M; Division of Pediatric Hematology, Oncology, Stem Cell Transplantation, University of Florida, Shands Children's Hospital, Gainesville, FL, USA., Castillo P; Division of Pediatric Hematology, Oncology, Stem Cell Transplantation, University of Florida, Shands Children's Hospital, Gainesville, FL, USA., Horn B; Division of Pediatric Hematology, Oncology, Stem Cell Transplantation, University of Florida, Shands Children's Hospital, Gainesville, FL, USA., Dotson N; Division of Pediatric Hematology, Oncology, Stem Cell Transplantation, University of Florida, Shands Children's Hospital, Gainesville, FL, USA., Moore-Higgs G; Division of Pediatric Hematology, Oncology, Stem Cell Transplantation, University of Florida, Shands Children's Hospital, Gainesville, FL, USA., Milner J; Division of Pediatric Hematology, Oncology, Stem Cell Transplantation, University of Florida, Shands Children's Hospital, Gainesville, FL, USA.
Jazyk:	angličtina
Zdroj:	Leukemia research reports [Leuk Res Rep] 2024 Jul 24; Vol. 22, pp. 100472. Date of Electronic Publication: 2024 Jul 24 (Print Publication: 2024).
DOI:	10.1016/j.lrr.2024.100472
Abstrakt:	Patients who receive allogeneic hematopoietic stem cell transplantation (alloHSCT) are at risk for developing persistent thrombocytopenia. Here, we describe treatment with avatrombopag, a thrombopoietin receptor agonist, in a pediatric patient with chronic, severe, transfusion-dependent thrombocytopenia (<10 × 10 3 /µL) post-alloHSCT that was persistent despite treatment with romiplostim, another thrombopoietin receptor agonist. Following the granting of a compassionate use investigational new drug authorization, avatrombopag treatment was initiated, and the patient's platelet count increased. To date, the patient has maintained a platelet count >100 × 10 3 /µL. No adverse events or medication toxicities have been reported, and he has resumed his pre-alloHSCT activities. Competing Interests: Avatrombopag was provided via compassionate use by Sobi, Inc. The authors have no conflicts of interest to declare related to the contents of this case study. (© 2024 Published by Elsevier Ltd.)
Databáze:	MEDLINE
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