Bone Marrow Transplantation for Patients with Myelodysplasia

Appelbaum, Frederick R.; Barrall, Janet; Storb, Rainer; Fisher, Lloyd D.; Schoch, Gary; Ramberg, R; Shulman, Howard M.; Anasetti, Claudio; Bearman, Scott I.; Beatty, Patrick G.; Bensinger, William I.; Cd, Buckner; Clift, Reginald A.; Hansen, John A.; Martin, Paul J.; Petersen, Finn; Sanders, Jean E.; Singer, Jack; Stewart, Patricia; Sullivan, Keith M.; Witherspoon, Robert P.; Ed, Thomas

doi:10.7326/0003-4819-112-8-590

Cited by 133 publications

(45 citation statements)

References 31 publications

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“…Age is usually an important prognostic factor for the outcome after various forms of transplantation. [11][12][13][14] However, age did not significantly affect the outcome of transplantation in this study. Younger patients were more often transplanted with mismatched donors and in advanced disease stages.…”

Section: Discussioncontrasting

confidence: 43%

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Genotypically nonidentical related donors for transplantation of patients with myelodysplastic syndromes: comparison with unrelated donor transplantation and autologous stem cell transplantation

Witte¹,

Pikkemaat²,

Hermans

et al. 2001

Leukemia

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Section: Discussioncontrasting

confidence: 43%

“…Younger age, shorter disease duration and a low percentage of marrow blasts (Ͻ5%) were associated with improved outcome. 11,12 However, most patients lack a genotypically identical donor. This analysis evaluated the results of transplantation with genotypically non-identical related donors.…”

Section: Discussionmentioning

confidence: 99%

Genotypically nonidentical related donors for transplantation of patients with myelodysplastic syndromes: comparison with unrelated donor transplantation and autologous stem cell transplantation

Witte¹,

Pikkemaat²,

Hermans

et al. 2001

Leukemia

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“…A total of 14 of the JMML patients have been previously reported, 18 and 29 of the patients with other subtypes of MDS have been previously included in analyses including adult MDS patients. [22][23][24] Analysis of prognostic factors and long-term survival demonstrates that bone marrow transplantation (BMT) is well tolerated and may be curative.…”

Section: Raeb; Raeb-t; Jmml; Cmmlmentioning

confidence: 99%

Allogeneic bone marrow transplantation in children with myelodysplastic syndrome or juvenile myelomonocytic leukemia: the Seattle experience

Yusuf

Frangoul

Gooley

et al. 2004

Bone Marrow Transplant

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Summary:The purpose of this study was to evaluate the role of allogeneic bone marrow transplantation (BMT) in children with myelodysplastic syndrome (MDS). In total, 94 consecutive pediatric patients with MDS received an allogeneic BMT from 1976 to 2001 for refractory anemia (RA) (n ¼ 25), RA with ringed sideroblasts (RARS) (n ¼ 2), RA with excess blasts (RAEB) (n ¼ 20), RAEB in transformation (RAEB-T) (n ¼ 14), juvenile myelomonocytic leukemia (JMML) (n ¼ 32) or chronic myelomonocytic leukemia (CMML) (n ¼ 1). The estimated 3-year probabilities of survival, event-free survival (EFS), nonrelapse mortality and relapse were 50, 41, 28 and 29%, respectively. Patients with RA/RARS had an estimated 3-year survival of 74% compared to 68% in those with RAEB and 33% in patients with JMML/CMML. In multivariable analysis, patients with RAEB-T or JMML were 3.9 and 3.7 times more likely to die compared to those with RA/RARS and RAEB (P ¼ 0.005 and 0.004, respectively). Patients with RAEB-T were 5.5 times more likely to relapse (P ¼ 0.01). The median follow-up among the 43 surviving patients is 10 years (range 1-25). We conclude that allogeneic BMT for children with MDS is well tolerated and can be curative.

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“…4 The only curative treatment for patients with MDS is allogeneic stem cell transplantation (SCT). [5][6][7][8] However, given the considerable morbidity and mortality associated with this approach, [9][10][11] candidate patients must be carefully selected. 12,13 All patients with MDS are likely to receive red blood cell transfusions at some point during their disease.…”

Section: Introductionmentioning

confidence: 99%

Prognostic impact of pre-transplantation transfusion history and secondary iron overload in patients with myelodysplastic syndrome undergoing allogeneic stem cell transplantation: a GITMO study

Alessandrino¹,

Porta²,

Bacigalupo³

et al. 2009

Haematologica

149

125

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BackgroundTransfusion-dependency affects the natural history of myelodysplastic syndromes. Secondary iron overload may concur to this effect. The relative impact of these factors on the outcome of patients with myelodysplastic syndrome receiving allogeneic stem-cell transplantation remains to be clarified. Design and MethodsWe retrospectively evaluated the prognostic effect of transfusion history and iron overload on the post-transplantation outcome of 357 patients with myelodysplastic syndrome reported to the Gruppo Italiano Trapianto di Midollo Osseo (GITMO) registry between 1997 and 2007. ResultsTransfusion-dependency was independently associated with reduced overall survival (hazard ratio=1.48, P=0.017) and increased non-relapse mortality (hazard ratio=1.68, P=0.024). The impact of transfusion-dependency was noted only in patients receiving myeloablative conditioning (overall survival: hazard ratio=1.76, P=0.003; non-relapse mortality: hazard ratio=1.70, P=0.02). There was an inverse relationship between transfusion burden and overall survival after transplantation (P=0.022); the outcome was significantly worse in subjects receiving more than 20 red cell units. In multivariate analysis, transfusion-dependency was found to be a risk factor for acute graft-versus-host disease (P=0.04). Among transfusion-dependent patients undergoing myeloablative allogeneic stem cell transplantation, pre-transplantation serum ferritin level had a significant effect on overall survival (P=0.01) and non-relapse mortality (P=0.03). This effect was maintained after adjusting for transfusion burden and duration, suggesting that the negative effect of transfusion history on outcome might be determined at least in part by iron overload. ConclusionsPre-transplantation transfusion history and serum ferritin have significant prognostic value in patients with myelodysplastic syndrome undergoing myeloablative allogeneic stem cell transplantation, inducing a significant increase of non-relapse mortality. These results indicate that transfusion history should be considered in transplantation decision-making in patients with myelodysplastic syndrome.

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Bone Marrow Transplantation for Patients with Myelodysplasia

Abstract: Bone marrow transplantation offers a potential cure for many patients with myelodysplasia. Best results can be expected in younger patients who receive transplants relatively early in their disease course.

Cited by 133 publications

References 31 publications

Genotypically nonidentical related donors for transplantation of patients with myelodysplastic syndromes: comparison with unrelated donor transplantation and autologous stem cell transplantation

Genotypically nonidentical related donors for transplantation of patients with myelodysplastic syndromes: comparison with unrelated donor transplantation and autologous stem cell transplantation

Allogeneic bone marrow transplantation in children with myelodysplastic syndrome or juvenile myelomonocytic leukemia: the Seattle experience

Prognostic impact of pre-transplantation transfusion history and secondary iron overload in patients with myelodysplastic syndrome undergoing allogeneic stem cell transplantation: a GITMO study

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