X-linked sideroblastic anemia in females

S Sarah Ducamp (1Department of Pathology, Boston Children’s Hospital and Harvard Medical School, Boston, MA) D Dean R. Campagna (1Department of Pathology, Boston Children’s Hospital and Harvard Medical School, Boston, MA) A Anoop K. Sendamarai (1Department of Pathology, Boston Children’s Hospital and Harvard Medical School, Boston, MA) P Paul J. Schmidt (1Department of Pathology, Boston Children’s Hospital and Harvard Medical School, Boston, MA) H Harrison K. Tsai (4Department of Pathology, Boston Children’s Hospital, Boston, MA) M Matthew M. Heeney (Dana–Farber/Boston Children’s Cancer and Blood Disorders Center, Boston) S Sylvia S. Bottomley (3Department of Medicine, University of Oklahoma College of Medicine, Oklahoma City, OK) M Mark D. Fleming

Abstract

Abstract X-linked sideroblastic anemia (XLSA) in female carriers of 5-aminolevulinic acid synthase 2 mutations is not uncommon. We describe unique features and genotype/phenotype correlations in females with XLSA and evaluate the contributions of X-chromosome skewing and clonal hematopoiesis, emphasizing the importance of distinguishing it from myelodysplastic syndromes with ring sideroblasts.

Article Details

Journal Blood
Volume / Issue Vol. 145, Issue 14
Published April 03, 2025
Pages 1583-1587
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (8)

S

Sarah Ducamp

1Department of Pathology, Boston Children’s Hospital and Harvard Medical School, Boston, MA

D

Dean R. Campagna

1Department of Pathology, Boston Children’s Hospital and Harvard Medical School, Boston, MA

A

Anoop K. Sendamarai

1Department of Pathology, Boston Children’s Hospital and Harvard Medical School, Boston, MA

P

Paul J. Schmidt

1Department of Pathology, Boston Children’s Hospital and Harvard Medical School, Boston, MA

H

Harrison K. Tsai

4Department of Pathology, Boston Children’s Hospital, Boston, MA

M

Matthew M. Heeney

Dana–Farber/Boston Children’s Cancer and Blood Disorders Center, Boston

S

Sylvia S. Bottomley

3Department of Medicine, University of Oklahoma College of Medicine, Oklahoma City, OK

M

Mark D. Fleming