Trilineage hematopoiesis induced by low-dose eltrombopag in a patient with Fanconi anemia can be used as a bridge to hematopoietic stem cell transplant

Ashish O Gupta, Rasmi Palassery, Howard Meyerson, Sanjay Ahuja, Yousif Matloub

Research output: Contribution to journalArticle

Abstract

Fanconi anemia (FA) is an autosomal recessive, progressive bone marrow failure disorder characterized by congenital defects and marked cancer predisposition. Hematopoietic stem cell transplant is the therapy of choice for FA patients with progressive pancytopenia. These patients receive multiple transfusions for cytopenias. Oxymetholone has been used with variable success to improve cytopenias. Eltrombopag has been shown to induce bilineage or trilineage hematopoiesis in aplastic anemia and patients with myelodysplastic marrow. We report a case of FA where eltrombopag in conjunction with oxymetholone induced trilineage hematopoiesis and eliminated transfusion requirement before transplant, thereby enhancing favorable outcome after hematopoietic stem cell transplant.

Original languageEnglish (US)
Pages (from-to)229-232
Number of pages4
JournalJournal of pediatric hematology/oncology
Volume41
Issue number3
DOIs
StatePublished - Apr 1 2019
Externally publishedYes

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Fanconi Anemia
Hematopoiesis
Oxymetholone
Hematopoietic Stem Cells
Transplants
Bone Marrow
Pancytopenia
Aplastic Anemia
eltrombopag
Neoplasms
Therapeutics

Keywords

  • Eltrombopag
  • Fanconi anemia
  • Trilineage hematopoiesis

PubMed: MeSH publication types

  • Journal Article

Cite this

Trilineage hematopoiesis induced by low-dose eltrombopag in a patient with Fanconi anemia can be used as a bridge to hematopoietic stem cell transplant. / Gupta, Ashish O; Palassery, Rasmi; Meyerson, Howard; Ahuja, Sanjay; Matloub, Yousif.

In: Journal of pediatric hematology/oncology, Vol. 41, No. 3, 01.04.2019, p. 229-232.

Research output: Contribution to journalArticle

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