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Hereditary sideroblastic anemia

MedGen UID:
65119
Concept ID:
C0221018
Disease or Syndrome
Synonyms: ANEMIA, SIDEROBLASTIC, X-LINKED; Inherited sideroblastic anemia
 
Monarch Initiative: MONDO:0020099
OMIM® Phenotypic series: PS300751
Orphanet: ORPHA98362

Definition

The essential features of X-linked sideroblastic anemia include the following: (1) a hypochromic microcytic anemia and 2 discrete populations of red blood cells, one microcytic and the other normocytic; (2) marrow ringed sideroblasts, particularly prominent in the late erythroid precursors; (3) a variable hematologic response to pharmacologic doses of pyridoxine; and (4) systemic iron overload secondary to chronic ineffective erythropoiesis. The age of clinical onset of the disorder can vary from in utero to the ninth decade. Whereas males are preferentially affected, females may present with clinically severe anemia. More commonly, female carriers of the disease have an increased red blood cell distribution width and sometimes erythrocyte dimorphism (Fleming, 2002). Genetic Heterogeneity of Sideroblastic Anemia See also SIDBA2 (205950), caused by mutation in the SLC25A38 gene (610819) on chromosome 3p22; SIDBA3 (616860), caused by mutation in the GLRX5 gene (609588) on chromosome 14q32; SIDBA4 (182170), caused by mutation in the HSPA9 gene (600548) on chromosome 5q31; and SIDBA5 (619523), caused by mutation in the HSCB gene (608142) on chromosome 22q12. [from OMIM]

Professional guidelines

PubMed

Meo A, Ruggeri A, La Rosa MA, Zanghì L, Morabito N, Duca L
Hemoglobin 2006;30(1):131-7. doi: 10.1080/03630260500455615. PMID: 16540426

Recent clinical studies

Etiology

Meo A, Ruggeri A, La Rosa MA, Zanghì L, Morabito N, Duca L
Hemoglobin 2006;30(1):131-7. doi: 10.1080/03630260500455615. PMID: 16540426
Furuyama K, Sassa S
Cell Mol Biol (Noisy-le-grand) 2002 Feb;48(1):5-10. PMID: 11929048
Aoki Y
J Clin Invest 1980 Jul;66(1):43-9. doi: 10.1172/JCI109833. PMID: 6249845Free PMC Article

Diagnosis

Meo A, Ruggeri A, La Rosa MA, Zanghì L, Morabito N, Duca L
Hemoglobin 2006;30(1):131-7. doi: 10.1080/03630260500455615. PMID: 16540426
Fairbanks VF, Dickson ER, Thompson ME
Hosp Pract (Off Ed) 1991 Apr;26 Suppl 3:53-5. doi: 10.1080/21548331.1991.11704290. PMID: 2010494
Aoki Y
J Clin Invest 1980 Jul;66(1):43-9. doi: 10.1172/JCI109833. PMID: 6249845Free PMC Article

Therapy

Meo A, Ruggeri A, La Rosa MA, Zanghì L, Morabito N, Duca L
Hemoglobin 2006;30(1):131-7. doi: 10.1080/03630260500455615. PMID: 16540426
Fairbanks VF, Dickson ER, Thompson ME
Hosp Pract (Off Ed) 1991 Apr;26 Suppl 3:53-5. doi: 10.1080/21548331.1991.11704290. PMID: 2010494
Soslau G, Brodsky I
Am J Hematol 1989 Dec;32(4):298-304. doi: 10.1002/ajh.2830320411. PMID: 2816925

Prognosis

Meo A, Ruggeri A, La Rosa MA, Zanghì L, Morabito N, Duca L
Hemoglobin 2006;30(1):131-7. doi: 10.1080/03630260500455615. PMID: 16540426

Clinical prediction guides

Aoki Y
J Clin Invest 1980 Jul;66(1):43-9. doi: 10.1172/JCI109833. PMID: 6249845Free PMC Article

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