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1362 Part X: Malignant Myeloid Diseases Chapter 87: Myelodysplastic Syndromes 1363

most patients are not candidates for any form of transplantation because 5. Parapia LA: Trepanning or trephines: A history of bone marrow biopsy. Br J Haematol
of advanced age or comorbid conditions. Outcomes are similar for those 139(1):14–19, 2007.
treated with myeloablative and reduced-intensity conditioning. For 6. Luzzatto AM: Sull’ anemia grave megaloblastica senza reporto ematologico corrispon-
559
dente (anemia pseudoaplastica). Riv veneta di sc med Venezia 47:193–212, 1907.
those patients who relapse after AHSCT, outcomes are grim. Salvage 7. di Guglielmo G: Eritremie acute. Boll Soc Med Chir 1:665–673, 1926.
therapy with donor lymphocyte infusions, second transplantations, or 8. Minot GR, Murphy WP: Treatment of pernicious anemia by a special diet. J Am Med
Assoc 87:470–476, 1926.
other immunologic manipulations may be feasible, but less than 10 per- 9. Rhoads CP, Barker WH: Refractory anemia: Analysis of 100 cases. JAMA 110:794–796,
cent of patients will experience prolonged disease-free survival. 560,561 1938.
10. Rosati S, Mick R, Xu F, et al: Refractory cytopenia with multilineage dysplasia: Fur-
Autologous Stem Cell Infusion ther characterization of an “unclassifiable” myelodysplastic syndrome. Leukemia 10(1):
20–26, 1996.
Patients with oligoblastic leukemia have been infused with their own 11. Chevallier P: Sur la terminologie des leucoses et les affections-frontieres: Les odoleu-
stem cells after intensive chemotherapy. 562–564 This strategy is rarely used coses. Sangre (Barc) 15:587–593, 1942–1943.
in the present era. In MDS, autologous transplantation is limited by con- 12. Hamilton-Paterson JL: Pre-leukaemic anaemia. Acta Haematol 2:309–316, 1949.
tamination of the stem cell product with a repopulating leukemic cell 13. Block M, Jacobson LO, Bethard WF: Preleukemic acute human leukemia. JAMA
152:1018–1028, 1953.
and the absence of a graft-versus-leukemia effect. In selected patients, 14. Bjorkman SE: Chronic refractory anemia with sideroblastic bone marrow: A study of
peritransplantation mortality with intensive therapy and stem cell four cases. Blood 11:250–259, 1956.
rescue has been approximately 10 percent, and approximately 50 per- 15. Saarni MI, Linman JW: Preleukemia. The hematologic syndrome preceding acute
leukemia. Am J Med 55(1):38–48, 1973.
cent of selected patients had extended survivals. The more advanced 16. Dreyfus B, Rochant H, Sultan C, et al: [Refractory anemia with excess myeloblasts in
565
the disease at the time of treatment, the worse the outcome. With the the bone marrow. Study of 11 cases]. Presse Med 78(8):359–364, 1970.
increasing use of reduced-intensity AHSCT, autologous stem cell trans- 17. Dreyfus B: Preleukemic states. I. Definition and classification. II. Refractory anemia
plantation has been used less often. An antecedent diagnosis of RAEB with an excess of myeloblasts in the bone marrow (smoldering acute leukemia). Nouv
Rev Fr Hematol Blood Cells 17(1–2):33–55, 1976.
had no influence on mobilization of blood stem cells and hematopoietic 18. Izrael V, Jacquillat C, Chastang C, et al: [New data about oligoblastic leukemias.
recovery after autologous stem cell transplantation for acute myeloid Apropos of an analysis of 120 cases]. Nouv Presse Med 4(13):947–952, 1975.
leukemia. 566 19. Bennett JM, Catovsky D, Daniel MT, et al: Proposals for the classification of the
acute leukaemias. French-American-British (FAB) co-operative group. Br J Haematol
33(4):451–458, 1976.
Other Cytotoxic Drugs 20. Galton DAG, Dacie, JV: Classification of the acute leukemias. Blood Cells 1:17–24, 1975.
Hydroxyurea and low-dose etoposide are useful in controlling leukemic 21. Bennett JM, Catovsky D, Daniel MT, et al: Proposals for the classification of the myelo-
dysplastic syndromes. Br J Haematol 51(2):189–199, 1982.
cell proliferation but usually produce only partial responses in higher-risk 22. Ma X: Epidemiology of myelodysplastic syndromes. Am J Med 125(7 Suppl):S2–S5,
MDS and do not influence survival duration. 567,568 Occasional patients 2012.
have achieved remissions with etoposide (50 mg as a 2-hour infusion, 23. Sekeres MA: The epidemiology of myelodysplastic syndromes. Hematol Oncol Clin
two to seven times weekly for 4 weeks; or 100 mg/day orally for 3 days North Am 24(2):287–294, 2010.
and then 50 mg twice weekly). Low-dose melphalan, gemcitabine, 24. Craig BM, Rollison DE, List AF, Cogle CR: Underreporting of myeloid malignancies by
570
568
569
United States cancer registries. Cancer Epidemiol Biomarkers Prev 21(3):474–481, 2012.
irinotecan (CPT-11), a DNA topoisomerase I inhibitor, troxacitabine, 25. Ma X, Does M, Raza A, Mayne ST: Myelodysplastic syndromes: Incidence and survival
571
574
an enantiomer of cytarabine, 572,573 and weekly doses of oral idarubicin in the United States. Cancer 109(8):1536–1542, 2007.
have each resulted in responses in some patients. Clofarabine, a purine 26. Cogle CR, Iannacone MR, Yu D, et al: High rate of uncaptured myelodysplastic syndrome
cases and an improved method of case ascertainment. Leuk Res 38(1):71–75, 2014.
nucleoside analogue, also has activity in MDS, although renal insuffi- 27. Cogle CR, Craig BM, Rollison DE, List AF: Incidence of the myelodysplastic syndromes
ciency and hepatotoxicity limit its use to patients who require cytore- using a novel claims-based algorithm: High number of uncaptured cases by cancer reg-
duction prior to transplant. 575 istries. Blood 117(26):7121–7125, 2011.
28. Visser O, Trama A, Maynadie M, et al: Incidence, survival and prevalence of myeloid
malignancies in Europe. Eur J Cancer 48(17):3257–3266, 2012.
Future Approaches 29. Gologan R: Epidemiological data on myelodysplastic syndrome patients under 50 years
The FDA has not approved any new drugs for MDS therapy since 2006, in a single center of Romania. Leuk Res 34(11):1442–1446, 2010.
and currently available therapies will lose effectiveness in the majority 30. Chen B, Zhao WL, Jin J, et al: Clinical and cytogenetic features of 508 Chinese patients
with myelodysplastic syndrome and comparison with those in Western countries.
of patients within 2 to 3 years after treatment initiation. Unfortunately, Leukemia 19(5):767–775, 2005.
targetable constitutively activating kinase mutations are rare in MDS, 31. Chatterjee T, Dixit A, Mohapatra M, et al: Clinical, haematological and histomorpho-
and for many MDS-associated mutations summarized above, including logical profile of adult myelodysplastic syndrome. Study of 96 cases in a single institute.
Eur J Haematol 73(2):93–97, 2004.
those that alter transcriptional regulation or pre-mRNA splicing, it is 32. Kuendgen A, Matsuda A, Germing U: Differences in epidemiology of MDS between
not clear how best to develop targeted therapy. In addition, clonal het- Western and Eastern countries: Ethnic differences or environmental influence? Leuk
erogeneity and the clonal architecture of MDS mean that currently it is Res 31(1):103–104, 2007.
often not known which mutations are early initiating events and which 33. Matsuda A, Germing U, Jinnai I, et al: Differences in the distribution of subtypes
according to the WHO classification 2008 between Japanese and German patients with
are later events important only for survival of a subclone. The advent refractory anemia according to the FAB classification in myelodysplastic syndromes.
576
of high-throughput techniques for genetic analysis and improved Leuk Res 34(8):974–980, 2010.
understanding of disease biology may lead to development of new, more 34. Ohba R, Furuyama K, Yoshida K, et al: Clinical and genetic characteristics of congenital
effective approaches in the future. sideroblastic anemia: Comparison with myelodysplastic syndrome with ring siderob-
last (MDS-RS). Ann Hematol 92(1):1–9, 2013.
35. Iwanaga M, Hsu WL, Soda M, et al: Risk of myelodysplastic syndromes in people
REFERENCES exposed to ionizing radiation: A retrospective cohort study of Nagasaki atomic bomb
survivors. J Clin Oncol 29(4):428–434, 2011.
36. Sekeres MA, Schoonen WM, Kantarjian H, et al: Characteristics of US patients with
1. Welch JS, Ley TJ, Link DC, et al: The origin and evolution of mutations in acute myeloid
leukemia. Cell 150(2):264–278, 2012. myelodysplastic syndromes: Results of six cross-sectional physician surveys. J Natl
2. Steensma DP: Historical perspectives on myelodysplastic syndromes. Leuk Res Cancer Inst 100(21):1542–1551, 2008.
36(12):1441–1452, 2012. 37. Breccia M, Mengarelli A, Mancini M, et al: Myelodysplastic syndromes in patients
3. Layton DM, Mufti GJ: Myelodysplastic syndromes: Their history, evolution and relation under 50 years old: A single institution experience. Leuk Res 29(7):749–754, 2005.
to acute myeloid leukaemia. Blut 53(6):423–436, 1986. 38. Stary J, Baumann I, Creutzig U, et al: Getting the numbers straight in pediatric MDS:
4. Lichtman MA: Does a diagnosis of myelogenous leukemia require 20% marrow mye- Distribution of subtypes after exclusion of down syndrome. Pediatr Blood Cancer
50(2):435–436, 2008.
loblasts, and does <5% marrow myeloblasts represent a remission? The history and 39. Niemeyer CM, Kratz CP, Hasle H: Pediatric myelodysplastic syndromes. Curr Treat
ambiguity of arbitrary diagnostic boundaries in the understanding of myelodysplasia.
Oncologist 18(9):973–980, 2013. Options Oncol 6(3):209–214, 2005.
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