Journal of Hematology - Supplements - Haematologica

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8 anemia of neonatal onset: a single-center retrospective study of six children. J Pediatr 1998; 132:599-612. 18. Chan GC, Head DR, Wang WC. Refractory anemia with ringed sideroblasts in children: two disease with a similar phenotype? J Pediatr Hematol Oncol 1999; 21:418-23. 19. Emanuel PD. Myelodysplasia and myeloprolipherative disorders in chidhood: an update. Br J Haematol 1999; 105:852-63. 20. Bader Meunier B, Tchernia G, Mielot F, et al. Occurrence of myeloproliferative disorder in patients with Noonan syndrome. J Pediatr 1997; 130:885-9. 21. Runde V, de Witte T, Arnold R, et al. Bone marrow transplantation from HLA-identical siblings as firstline treatment in patients with MDS: early transplantation is associated with improved outcome. Chronic Leukemia Working Party of European Group for Blood and Marrow transplantation. Bone Marrow Transplant 1998; 21:255-61. 22. Hasle H, Arico M, Basso G, et al. Myelodysplastic syndrome, juvenile myelomonocytic leukaemia, and AML associated with complete or partial monosomy 7. European Working Group on MDS in childhood (EWOG-MDS). Leukemia 1999; 13:376-85. 23. Pitcher LA, Hann IM, Evans JP, Veys P, Chessells JM, Webb DK. Improved prognosis for acquired aplastic anemia. Arch Dis Child 1999; 80:158-62. 24. Ball SE. The modern management of SAA. Br J Haematol 2000; 110:41-53. 25. Heimpel H. Aplastic anemia before BMT and ATG. Acta Haematol 2000; 103:11-5. 26. Bacigalupo A, Oneto R, Bruno B, et al. Current results of bone marrow transplantation in patients with acquired severe aplastic anemia. Report of the European Group for Blood and Marrow transplantation. On behalf of the Working Party on Severe Aplastic Anemia of the European Group for Blood and Marrow Transplantation. Acta Haematol 2000; 103:19-25. 27. Gluckman E, Esperou-Bourdeau H, Baruchel A, et al. Multicenter randomized study comparing cyclosporin- A alone and ATG with prednisolone for treatment of severe aplastic anemia. Blood 1992; 79:2540-6. 28. Giardini C, Lucarelli G. Bone marrow transplantation for beta-thalassemia. Hematol Oncol Clin North Am 1999; 13:1059-64. 29. Piga A, Longo F, Voi V, Facello S, Miniero R, Dresow B. Late effects of bone marrow transplantation for thalassemia. Ann N Y Acad Sci 1998; 850:294-9. 30. Lucarelli G, Galimberti M, Giardini C, et al. Bone marrow transplantation in thalassemia. The experience of Pesaro. Ann N Y Acad Sci 1998; Jun 30; 850:270-5. 31. Gaziev D, Polchi P, Lucarelli G, et al. Second marrow transplants for graft failure in patients with Thalassemia. Bone Marrow Transplant 1999; 24:1299- 306. 32. Li CK, Lai DH, Shing MM, Chik KW, Lee V, Yuen PM. Early iron reduction programme for Thalassemia patients after BMT. Bone Marrow Transplant 2000; 25:653-6. 33. Nietert PJ, Abboud MR, Silverstein MD, Jackson SM. Bone marrow transplantation versus periodic prophylactic blood transfusion in sickle cell patients at high risk of ischemic stroke: a decision analysis. Blood 2000; 15; 95:3057-64. 34. Walters MC, Storb R, Patience M, et al. Impact of BMT for symptomatic sickle cell disease: an interim report. Blood 2000; 95:1918-24. 35. Walters MC, Patience M, Leisenring W, et al. Barriers to bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant 1996; 2:100-4. 36. Bernaudin F. Results and current indications of bone marrow allograft in sickle cell disease. Pathol Biol 1999; 47:59-64. haematologica vol. 85(supplement to n. 11):November 2000
haematologica 2000; 85(supplement to n. 11):9-11 original paper Indications and role of allogeneic bone marrow transplantation in childhood very high risk acute lymphoblastic leukemia in first complete remission CORNELIO UDERZO BMT Unit, Pediatric Hematologic Department, S.Gerardo Hospital, Monza, University of Milano Bicocca, Italy ABSTRACT Background and Objectives. Acute lymphoblastic leukemia (ALL) accounts for approximately one third of all cancers in children and its outcome depends on risk factors at the time of diagnosis. While uniform chemotherapy adopted in multicenter studies provided a constant improvement in cure rates for standard risk patients, the results reached in very high risk patients have been disappointing. The objective of this review is to point out the role of allogeneic bone marrow transplantation (alloBMT) in very high risk childhood ALL on the basis of results from the current clinical trials. Evidence and information source. Data covered by Medline and produced by the authors involved in ongoing international studies cover a vast “scenario” of children with very high risk ALL who underwent allogeneic BMT. State of art. The author outlines the crucial point of very high risk factors in childhood ALL in order to identify those children who are at risk of early relapse. The main reasons for pursuing alloBMT in this particular category of patients concern poor prognostic factors such as molecular biology markers, structural chromosomal abnormalities and biological factors (poor prednisone response) including resistance to initial induction chemotherapy. AlloBMT in childhood ALL in first complete remission seemed to lead to a promising disease-free survival in this patient population when compared with chemotherapy. The principal biases of the retrospective studies were the variable very high risk eligibility criteria, the different first-line therapies adopted before alloBMT and above all the waiting time to transplant which could have accounted for some advantage to alloBMT patients versus chemotherapy patients. Perspectives. The author touches upon the preliminary results of an ongoing international prospective study as an example of reaching a consensus in the controversial treatment of childhood very high risk Correspondence: Cornelio Uderzo, Centro TMO, Clinica Pediatrica, Ospedale S.Gerardo di Monza,Università di Milano Bicocca, via Donizetti 106, 20052 Monza (MI), Italy. Phone and Fax: international +39-039-2332442 – E-Mail: ctmomonza@libero.it ALL. This attempt should provide more information regarding the role of alloBMT in this setting and should cover an area of particular interest. ©2000, Ferrata Storti Foundation Key words: ABMT, acute lymphoblastic leukemia In the last decade many data have emerged from single institute or multicenter trials involving children affected by very high risk ALL in 1 st complete remission (CR), but variable results have been shown regarding the eventfree survival (EFS) which ranges from 50% to 70% in patients treated with alloBMT 1-6 and from 10 to 40% in those treated with chemotherapy. 6-11 These data primarily depend on the selection of the patients on the basis of very high risk eligibility criteria, the intensity of the first-line treatment and subsequently the quality of the bone marrow remission. Current definition of very high risk factors in childhood ALL One of the crucial points explaining different results obtained by different strategies is the definition of the very high risk criteria over the years. International co-operative groups (POG, CCG, I-BFM-SG, AIEOP ) are using a similar risk classification scheme including the following very high risk criteria in the ongoing trials: a) genetic features (MLL rearrangements and BCR ABL fusion which affect less than 10 % of children with ALL), b) hyperleukocytosis (white blood cell count ≥ 100,000/mm 3 ) and/or T- immunophenotype combined with poor prednisone response, c) induction failure and, more recently, d) minimal residual disease (MRD) persistence. Other previous risk factors such as myeloid markers, poor prednisone response per se, hyperleukocytosis or T-immunophenotype alone, are no longer considered poor prognosis indicators. 11-15 haematologica vol. 85(supplement to n. 11):November 2000
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