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Hemopoietic cell transplantation for the myelodysplastic syndromes

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Scott B. L., Deeg H. J.
Archivum Immunologiae et Therapiae Experimentalis
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2005
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vol. 53
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issue 4
297-307
EN
Myelodysplastic syndromes (MDS) are hemopoietic stem cell disorders, and hemopoietic stem cell transplantation is currently the only therapeutic modality with curative potential. Among patients with less advanced/low-risk MDS (<5% marrow blasts), 3-year survivals of 65?70% are achievable with HLA-identical related and unrelated donors. The overall probability of disease recurrence in these patients is <5%. Among patients with more advanced disease (5% marrow blasts), the relapse probability is higher, ranging from 10?40%, and relapse-free survival is correspondingly lower. The criteria proposed by the International Prognostic Scoring System, derived from non-transplanted patients, also predict survival following transplantation. The development of reduced-intensity conditioning regimens and modification of conventional regimens, all aimed at optimizing the transplant approach, have permitted successful hemopoietic stem cell transplants even in patients 60?70 years of age. Improved survival with transplants from unrelated volunteer donors reflects to a large extent selection of donors on the basis of high resolution (allele-level) HLA typing. Graft-versus-host disease and associated problems remain major challenges after allogeneic transplantation. Autologous stem cell transplantation may be beneficial for selected patients who have obtained complete remissions with conventional chemotherapy.
2

Hematopoietic cell transplantation for chronic myeloproliferative disorders

100%
Tse W., Deeg H. J.
Archivum Immunologiae et Therapiae Experimentalis
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2006
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vol. 54
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issue 6
375-380
EN
Myeloproliferative disorders, including chronic idiopathic myelofibrosis (CIMF), polycythemia vera (PV), essential thrombocythemia (ET), and chronic myelomonocytic leukemia (CMML), are clonal diseases of hematopoietic stem or precursor cells. They often show a protracted or chronic course; however, all have the potential of progressing to severe marrow failure, associated with myelofibrosis, or of transforming into acute leukemia. At that point, hematopoietic cell transplantation (HCT) is the only current treatment strategy with curative potential. If transplantation is being considered and a suitable donor is available, HCT should be carried out before leukemic transformation has occurred, as the success rate of HCT declines steeply in patients who have evolved to leukemia. As many as 75?80% of patients with the original diagnoses of PV or ET, about 65?70% with CIMF, and 45% of patients with CMML are surviving long term after allogeneic HCT using conventional transplant regimens, with follow-up now extending to 15 years. Results with HLA-identical related and unrelated donors are comparable. Major risk factors for the outcome after HCT are the disease stage, the presence of comorbid conditions, and patient age. The development of reduced-intensity conditioning regimens has allowed for successful HCT even for older patients and patients with comorbid conditions. Studies on disease mechanisms, including the recent characterization of an activating mutation in JAK2, may provide additional prognostic guidance and are likely to lead to the development of novel treatment strategies, which will require continuous reassessment as to the optimum timing of HCT.
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