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Refractory anemia with excess blasts in transformation

MedGen UID:
124692
•Concept ID:
C0280028
•
Disease or Syndrome
Synonyms: RAEB-t; Refractory Anemia with Excess Blasts in Transformation; refractory anemia with excess blasts in transformation
 
Monarch Initiative: MONDO:0015692
Orphanet: ORPHA168960

Definition

A rare hematologic disease characterized by the presence of 20-29% blasts in the bone marrow, presence of 5-29% blasts in the peripheral blood, and/or presence of Auer rods. Patients show relatively stable peripheral blood counts for weeks or months, with specific cytogenetic and molecular genetic characteristics constituting important prognostic factors. [from ORDO]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVRefractory anemia with excess blasts in transformation
Follow this link to review classifications for Refractory anemia with excess blasts in transformation in Orphanet.

Professional guidelines

PubMed

Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure.
Prébet T, Gore SD, Esterni B, Gardin C, Itzykson R, Thepot S, Dreyfus F, Rauzy OB, Recher C, Adès L, Quesnel B, Beach CL, Fenaux P, Vey N
J Clin Oncol 2011 Aug 20;29(24):3322-7. Epub 2011 Jul 25 doi: 10.1200/JCO.2011.35.8135. PMID: 21788559Free PMC Article
New agents for the treatment of acute myelogenous leukemia: focus on topotecan and retinoids.
Estey EH
Leukemia 1998 Sep;12 Suppl 1:S13-5. PMID: 9777888
Myelodysplastic syndromes in the elderly: the role of growth factors in management.
Saba HI
Leuk Res 1996 Mar;20(3):203-19. doi: 10.1016/0145-2126(95)00131-x. PMID: 8637215

Recent clinical studies

Etiology

Myelodysplastic and myeloproliferative disorders of childhood.
Hasle H
Hematology Am Soc Hematol Educ Program 2016 Dec 2;2016(1):598-604. doi: 10.1182/asheducation-2016.1.598. PMID: 27913534Free PMC Article
Decitabine versus best supportive care in older patients with refractory anemia with excess blasts in transformation (RAEBt) - results of a subgroup analysis of the randomized phase III study 06011 of the EORTC Leukemia Cooperative Group and German MDS Study Group (GMDSSG).
Becker H, Suciu S, Rüter BH, Platzbecker U, Giagounidis A, Selleslag D, Labar B, Germing U, Salih HR, Muus P, Pflüger KH, Hagemeijer A, Schaefer HE, Fiaccadori V, Baron F, Ganser A, Aul C, de Witte T, Wijermans PW, Lübbert M
Ann Hematol 2015 Dec;94(12):2003-13. Epub 2015 Sep 24 doi: 10.1007/s00277-015-2489-6. PMID: 26400023
Classification of myeloid neoplasms: a comparative review.
McManus PM
Vet Clin Pathol 2005 Sep;34(3):189-212. doi: 10.1111/j.1939-165x.2005.tb00042.x. PMID: 16134066
Refractory anemia with excess of blasts in transformation: analysis of reclassification according to the WHO proposals.
Strupp C, Gattermann N, Giagounidis A, Aul C, Hildebrandt B, Haas R, Germing U
Leuk Res 2003 May;27(5):397-404. doi: 10.1016/s0145-2126(02)00220-5. PMID: 12620291
Childhood myelodysplasia.
Agarwal BR, Currimbhoy ZE
Indian Pediatr 1994 Jul;31(7):797-806. PMID: 7890342

Diagnosis

Myelodysplastic and myeloproliferative disorders of childhood.
Hasle H
Hematology Am Soc Hematol Educ Program 2016 Dec 2;2016(1):598-604. doi: 10.1182/asheducation-2016.1.598. PMID: 27913534Free PMC Article
Classification of myeloid neoplasms: a comparative review.
McManus PM
Vet Clin Pathol 2005 Sep;34(3):189-212. doi: 10.1111/j.1939-165x.2005.tb00042.x. PMID: 16134066
Comparison of idarubicin + ara-C-, fludarabine + ara-C-, and topotecan + ara-C-based regimens in treatment of newly diagnosed acute myeloid leukemia, refractory anemia with excess blasts in transformation, or refractory anemia with excess blasts.
Estey EH, Thall PF, Cortes JE, Giles FJ, O'Brien S, Pierce SA, Wang X, Kantarjian HM, Beran M
Blood 2001 Dec 15;98(13):3575-83. doi: 10.1182/blood.v98.13.3575. PMID: 11739159
Splenic pathology in myelodysplasia: a report of 13 cases with clinical correlation.
Kraus MD, Bartlett NL, Fleming MD, Dorfman DM
Am J Surg Pathol 1998 Oct;22(10):1255-66. doi: 10.1097/00000478-199810000-00011. PMID: 9777988
Childhood myelodysplasia.
Agarwal BR, Currimbhoy ZE
Indian Pediatr 1994 Jul;31(7):797-806. PMID: 7890342

Therapy

Decitabine versus best supportive care in older patients with refractory anemia with excess blasts in transformation (RAEBt) - results of a subgroup analysis of the randomized phase III study 06011 of the EORTC Leukemia Cooperative Group and German MDS Study Group (GMDSSG).
Becker H, Suciu S, Rüter BH, Platzbecker U, Giagounidis A, Selleslag D, Labar B, Germing U, Salih HR, Muus P, Pflüger KH, Hagemeijer A, Schaefer HE, Fiaccadori V, Baron F, Ganser A, Aul C, de Witte T, Wijermans PW, Lübbert M
Ann Hematol 2015 Dec;94(12):2003-13. Epub 2015 Sep 24 doi: 10.1007/s00277-015-2489-6. PMID: 26400023
Comparison of idarubicin + ara-C-, fludarabine + ara-C-, and topotecan + ara-C-based regimens in treatment of newly diagnosed acute myeloid leukemia, refractory anemia with excess blasts in transformation, or refractory anemia with excess blasts.
Estey EH, Thall PF, Cortes JE, Giles FJ, O'Brien S, Pierce SA, Wang X, Kantarjian HM, Beran M
Blood 2001 Dec 15;98(13):3575-83. doi: 10.1182/blood.v98.13.3575. PMID: 11739159
Low-dose cytarabine and aclarubicin in combination with granulocyte colony-stimulating factor (CAG regimen) for previously treated patients with relapsed or primary resistant acute myelogenous leukemia (AML) and previously untreated elderly patients with AML, secondary AML, and refractory anemia with excess blasts in transformation.
Saito K, Nakamura Y, Aoyagi M, Waga K, Yamamoto K, Aoyagi A, Inoue F, Nakamura Y, Arai Y, Tadokoro J, Handa T, Tsurumi S, Arai H, Kawagoe Y, Gunnji H, Kitsukawa Y, Takahashi W, Furusawa S
Int J Hematol 2000 Apr;71(3):238-44. PMID: 10846828
New agents for the treatment of acute myelogenous leukemia: focus on topotecan and retinoids.
Estey EH
Leukemia 1998 Sep;12 Suppl 1:S13-5. PMID: 9777888
Myelodysplastic syndromes in the elderly: the role of growth factors in management.
Saba HI
Leuk Res 1996 Mar;20(3):203-19. doi: 10.1016/0145-2126(95)00131-x. PMID: 8637215

Prognosis

Decitabine versus best supportive care in older patients with refractory anemia with excess blasts in transformation (RAEBt) - results of a subgroup analysis of the randomized phase III study 06011 of the EORTC Leukemia Cooperative Group and German MDS Study Group (GMDSSG).
Becker H, Suciu S, Rüter BH, Platzbecker U, Giagounidis A, Selleslag D, Labar B, Germing U, Salih HR, Muus P, Pflüger KH, Hagemeijer A, Schaefer HE, Fiaccadori V, Baron F, Ganser A, Aul C, de Witte T, Wijermans PW, Lübbert M
Ann Hematol 2015 Dec;94(12):2003-13. Epub 2015 Sep 24 doi: 10.1007/s00277-015-2489-6. PMID: 26400023
Refractory anemia with excess of blasts in transformation: analysis of reclassification according to the WHO proposals.
Strupp C, Gattermann N, Giagounidis A, Aul C, Hildebrandt B, Haas R, Germing U
Leuk Res 2003 May;27(5):397-404. doi: 10.1016/s0145-2126(02)00220-5. PMID: 12620291
Comparison of idarubicin + ara-C-, fludarabine + ara-C-, and topotecan + ara-C-based regimens in treatment of newly diagnosed acute myeloid leukemia, refractory anemia with excess blasts in transformation, or refractory anemia with excess blasts.
Estey EH, Thall PF, Cortes JE, Giles FJ, O'Brien S, Pierce SA, Wang X, Kantarjian HM, Beran M
Blood 2001 Dec 15;98(13):3575-83. doi: 10.1182/blood.v98.13.3575. PMID: 11739159
Splenic pathology in myelodysplasia: a report of 13 cases with clinical correlation.
Kraus MD, Bartlett NL, Fleming MD, Dorfman DM
Am J Surg Pathol 1998 Oct;22(10):1255-66. doi: 10.1097/00000478-199810000-00011. PMID: 9777988
Childhood myelodysplasia.
Agarwal BR, Currimbhoy ZE
Indian Pediatr 1994 Jul;31(7):797-806. PMID: 7890342

Clinical prediction guides

Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure.
Prébet T, Gore SD, Esterni B, Gardin C, Itzykson R, Thepot S, Dreyfus F, Rauzy OB, Recher C, Adès L, Quesnel B, Beach CL, Fenaux P, Vey N
J Clin Oncol 2011 Aug 20;29(24):3322-7. Epub 2011 Jul 25 doi: 10.1200/JCO.2011.35.8135. PMID: 21788559Free PMC Article
Refractory anemia with excess of blasts in transformation: analysis of reclassification according to the WHO proposals.
Strupp C, Gattermann N, Giagounidis A, Aul C, Hildebrandt B, Haas R, Germing U
Leuk Res 2003 May;27(5):397-404. doi: 10.1016/s0145-2126(02)00220-5. PMID: 12620291
Immunophenotypic clustering of myelodysplastic syndromes.
Maynadié M, Picard F, Husson B, Chatelain B, Cornet Y, Le Roux G, Campos L, Dromelet A, Lepelley P, Jouault H, Imbert M, Rosenwadj M, Vergé V, Bissières P, Raphaël M, Béné MC, Feuillard J; Groupe d'Etude Immunologique des Leucémies (GEIL)
Blood 2002 Oct 1;100(7):2349-56. doi: 10.1182/blood-2002-01-0230. PMID: 12239142
Intensive chemotherapy for patients with high-risk myelodysplastic syndrome.
Beran M
Int J Hematol 2000 Aug;72(2):139-50. PMID: 11039661
Solid and hematologic malignancies in 60 patients with systemic mast cell disease.
Travis WD, Li CY, Bergstralh EJ
Arch Pathol Lab Med 1989 Apr;113(4):365-8. PMID: 2650653

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