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Refractory anemia with ringed sideroblasts(RARS)

MedGen UID:
220394
Concept ID:
C1264195
Neoplastic Process
Synonyms: Pyridoxine refractory sideroblastic anemia; RARS; Sideroblastic anemia pyridoxine-refractory autosomal recessive
 
HPO: HP:0004828

Definition

A type of myelodysplastic syndrome characterized by less than 5% myeloblasts in the bone marrow, but with 15% or greater red cell precursors in the marrow being abnormal iron-stuffed cells called ringed sideroblasts. [from HPO]

Conditions with this feature

Autosomal dominant sideroblastic anemia
MedGen UID:
902781
Concept ID:
C4225428
Disease or Syndrome
Sideroblastic anemia comprises a heterogeneous group of inherited and acquired disorders characterized by ineffective erythropoiesis. Anemia, if present, may be microcytic or macrocytic. Sometimes a dimorphic picture is observed in which 2 populations of erythrocytes can be detected in peripheral blood smears. The presence of ringed sideroblasts (erythroblasts containing pathologic mitochondrial iron deposits) in bone marrow is pathognomonic for sideroblastic anemia (van Waveren Hogervorst et al., 1987; Schmitz-Abe et al., 2015). For a discussion of genetic heterogeneity of sideroblastic anemia, see SIDBA1 (300751).
Erythroleukemia, familial, susceptibility to
MedGen UID:
1790819
Concept ID:
C5552985
Finding
Familial erythroleukemia (FERLK) is a leukemic or preleukemic state in which red cell proliferation is the predominant feature. Hematologic characteristics include particularly ineffective and hyperplastic erythropoiesis with megaloblastic components accompanied by myeloblastic proliferation of varying degree (Park et al., 2002). Park et al. (2002) discussed the evolution of the definition of 'erythroleukemia,' which is considered by most to be a subtype of acute myelogenous leukemia (AML; 601626). Controversy about the precise definition of erythroleukemia revolves around the number or percentage of erythroblasts and myeloblasts found in the bone marrow and peripheral circulation. In the French-American-British (FAB) classification system (Bennett et al., 1985), it is known as AML-M6, whereas in the revised World Health Organization (WHO) classification system (Harris et al., 1999), it is known as 'AML, not otherwise categorized' (Zini and D'Onofrio, 2004).
Anemia, sideroblastic, 5
MedGen UID:
1794195
Concept ID:
C5561985
Disease or Syndrome
Sideroblastic anemia-5 (SIDBA5) is an autosomal recessive hematologic disorder characterized by abnormal iron accumulation in the mitochondria or erythroid cells. The pathologic iron deposits appear to ring the nucleus, resulting in a 'ringed sideroblast' on pathologic examination. Affected individuals have congenital hypochromic microcytic anemia apparent in childhood; they may also develop thrombocytopenia or pancytopenia (summary by Crispin et al., 2020). For a discussion of genetic heterogeneity of sideroblastic anemia, see SIDBA1 (300751).

Professional guidelines

PubMed

Onida F, Brand R, van Biezen A, Schaap M, von dem Borne PA, Maertens J, Beelen DW, Carreras E, Alessandrino EP, Volin L, Kuball JH, Figuera A, Sierra J, Finke J, Kröger N, de Witte T; MDS subcommittee of the EBMT-CMWP
Haematologica 2014 Oct;99(10):1582-90. Epub 2014 Aug 1 doi: 10.3324/haematol.2014.106880. PMID: 25085359Free PMC Article
Raptis A, Duh MS, Wang ST, Dial E, Fanourgiakis I, Fortner B, Paley C, Mody-Patel N, Corral M, Scott J
Transfusion 2010 Jan;50(1):190-9. Epub 2009 Aug 28 doi: 10.1111/j.1537-2995.2009.02361.x. PMID: 19719471
Buzaid AC, Garewal HS, Greenberg BR
Am J Med 1986 Jun;80(6):1149-57. doi: 10.1016/0002-9343(86)90677-7. PMID: 3524212

Recent clinical studies

Etiology

Ou R, Huang J, Shen H, Liu Z, Zhu Y, Zhong Q, Zheng L, Yao M, She Y, Zhou S, Chen R, Li C, Zhang Q, Liu S
Leuk Res 2017 Nov;62:40-50. Epub 2017 Sep 8 doi: 10.1016/j.leukres.2017.09.003. PMID: 28982058
Hlaváčková A, Štikarová J, Pimková K, Chrastinová L, Májek P, Kotlín R, Čermák J, Suttnar J, Dyr JE
Free Radic Biol Med 2017 Jul;108:1-7. Epub 2017 Mar 12 doi: 10.1016/j.freeradbiomed.2017.03.007. PMID: 28300669
Malcovati L, Della Porta MG, Pietra D, Boveri E, Pellagatti A, Gallì A, Travaglino E, Brisci A, Rumi E, Passamonti F, Invernizzi R, Cremonesi L, Boultwood J, Wainscoat JS, Hellström-Lindberg E, Cazzola M
Blood 2009 Oct 22;114(17):3538-45. Epub 2009 Aug 19 doi: 10.1182/blood-2009-05-222331. PMID: 19692701
Björk J, Albin M, Mauritzson N, Strömberg U, Johansson B, Hagmar L
Epidemiology 2000 May;11(3):285-91. doi: 10.1097/00001648-200005000-00010. PMID: 10784245
Nand S, Godwin JE
Cancer 1988 Sep 1;62(5):958-64. doi: 10.1002/1097-0142(19880901)62:5<958::aid-cncr2820620519>3.0.co;2-p. PMID: 3409176

Diagnosis

Hlaváčková A, Štikarová J, Pimková K, Chrastinová L, Májek P, Kotlín R, Čermák J, Suttnar J, Dyr JE
Free Radic Biol Med 2017 Jul;108:1-7. Epub 2017 Mar 12 doi: 10.1016/j.freeradbiomed.2017.03.007. PMID: 28300669
Malcovati L, Della Porta MG, Pietra D, Boveri E, Pellagatti A, Gallì A, Travaglino E, Brisci A, Rumi E, Passamonti F, Invernizzi R, Cremonesi L, Boultwood J, Wainscoat JS, Hellström-Lindberg E, Cazzola M
Blood 2009 Oct 22;114(17):3538-45. Epub 2009 Aug 19 doi: 10.1182/blood-2009-05-222331. PMID: 19692701
Björk J, Albin M, Mauritzson N, Strömberg U, Johansson B, Hagmar L
Epidemiology 2000 May;11(3):285-91. doi: 10.1097/00001648-200005000-00010. PMID: 10784245
Chan GC, Head DR, Wang WC
J Pediatr Hematol Oncol 1999 Sep-Oct;21(5):418-23. doi: 10.1097/00043426-199909000-00015. PMID: 10524457
Nand S, Godwin JE
Cancer 1988 Sep 1;62(5):958-64. doi: 10.1002/1097-0142(19880901)62:5<958::aid-cncr2820620519>3.0.co;2-p. PMID: 3409176

Therapy

Morita Y, Maeda Y, Yamaguchi T, Urase F, Kawata S, Hanamoto H, Tsubaki K, Ishikawa J, Shibayama H, Matsumura I, Matsuda M
Cancer Sci 2018 Oct;109(10):3209-3215. Epub 2018 Aug 26 doi: 10.1111/cas.13739. PMID: 30007103Free PMC Article
Howe RB, Porwit-MacDonald A, Wanat R, Tehranchi R, Hellström-Lindberg E
Blood 2004 May 1;103(9):3265-70. Epub 2003 Dec 18 doi: 10.1182/blood-2003-06-2124. PMID: 14684416
Kaur A, Yu SS, Lee AJ, Chiao TB
Ann Pharmacother 2003 Jul-Aug;37(7-8):1040-3. doi: 10.1345/aph.1D003. PMID: 12841816
Litvak D, Kirsner RS, Pakdaman NN, Federman DG
South Med J 2000 Sep;93(9):923-5. PMID: 11005358
Buzaid AC, Garewal HS, Greenberg BR
Am J Med 1986 Jun;80(6):1149-57. doi: 10.1016/0002-9343(86)90677-7. PMID: 3524212

Prognosis

Morita Y, Maeda Y, Yamaguchi T, Urase F, Kawata S, Hanamoto H, Tsubaki K, Ishikawa J, Shibayama H, Matsumura I, Matsuda M
Cancer Sci 2018 Oct;109(10):3209-3215. Epub 2018 Aug 26 doi: 10.1111/cas.13739. PMID: 30007103Free PMC Article
Yoshimi A, Abdel-Wahab O
Int J Hematol 2017 Jun;105(6):720-731. Epub 2017 May 2 doi: 10.1007/s12185-017-2242-0. PMID: 28466384
Hlaváčková A, Štikarová J, Pimková K, Chrastinová L, Májek P, Kotlín R, Čermák J, Suttnar J, Dyr JE
Free Radic Biol Med 2017 Jul;108:1-7. Epub 2017 Mar 12 doi: 10.1016/j.freeradbiomed.2017.03.007. PMID: 28300669
Chan GC, Head DR, Wang WC
J Pediatr Hematol Oncol 1999 Sep-Oct;21(5):418-23. doi: 10.1097/00043426-199909000-00015. PMID: 10524457
Nand S, Godwin JE
Cancer 1988 Sep 1;62(5):958-64. doi: 10.1002/1097-0142(19880901)62:5<958::aid-cncr2820620519>3.0.co;2-p. PMID: 3409176

Clinical prediction guides

Yoshimi A, Abdel-Wahab O
Int J Hematol 2017 Jun;105(6):720-731. Epub 2017 May 2 doi: 10.1007/s12185-017-2242-0. PMID: 28466384
Hlaváčková A, Štikarová J, Pimková K, Chrastinová L, Májek P, Kotlín R, Čermák J, Suttnar J, Dyr JE
Free Radic Biol Med 2017 Jul;108:1-7. Epub 2017 Mar 12 doi: 10.1016/j.freeradbiomed.2017.03.007. PMID: 28300669
Malcovati L, Della Porta MG, Pietra D, Boveri E, Pellagatti A, Gallì A, Travaglino E, Brisci A, Rumi E, Passamonti F, Invernizzi R, Cremonesi L, Boultwood J, Wainscoat JS, Hellström-Lindberg E, Cazzola M
Blood 2009 Oct 22;114(17):3538-45. Epub 2009 Aug 19 doi: 10.1182/blood-2009-05-222331. PMID: 19692701
Howe RB, Porwit-MacDonald A, Wanat R, Tehranchi R, Hellström-Lindberg E
Blood 2004 May 1;103(9):3265-70. Epub 2003 Dec 18 doi: 10.1182/blood-2003-06-2124. PMID: 14684416
Novaretti MC, Sopelete CR, Velloso ER, Rosa MF, Dorlhiac-Llacer PE, Chamone DA
Acta Haematol 2001;105(1):1-6. doi: 10.1159/000046525. PMID: 11340246

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