Comparison between bone marrow transplantation and antithymocyte globulin in treatment of young patients with severe aplastic anemia

Bayever, Eliel; Champlin, Richard E.; Ho, Winston G.; Lenarsky, Carl; Storch, Susan K.; Ladisch, Stephan; Gale, Robert Peter; Feig, Stephen A.

doi:10.1016/s0022-3476(84)80078-5

Cited by 40 publications

(24 citation statements)

References 27 publications

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“…Consistent with the EBMT studies, 10,22 the survival of children with SAA initially treated with IST has improved markedly since the 1980s, when first-line IST gave greatly inferior survival (with overall survival rates of around 40-50%) when compared with first-line BMT 1,5,6,20,21 ; in the current analyses, the probability of overall survival at 10 years in the patients treated first-line with IST reached 88%, which was comparable to that of the group treated first-line with BMT. Recent significant advances in secondline SCT, especially with a matched unrelated donor, may contribute to this marked improvement in survival after first-line IST.…”

Section: Discussionmentioning

confidence: 70%

“…The current guideline recommends BMT from an MFD as the treatment of choice for pediatric SAA [17][18][19] based on the results of comparative studies performed in the 1980s. 1,5,6,20,21 On the other hand, recent prospective studies with intensified IST for pediatric SAA have resulted in dramatic improvements in survival. 22,23 For example, a study from the EBMT showed a 100% overall survival rate at 6 years after first-line IST in 31 SAA patients younger than 20 years treated from 2002 to 2008.…”

Section: Discussionmentioning

confidence: 99%

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First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy

Yoshida¹,

et al. 2014

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ABSTRACThad a high risk of bias due to their study design and were conducted more than 10 years ago and may not be applicable to the standard of care of today.11 Updated evidence to aid treatment decisions in pediatric SAA is, therefore, required.In children, the choice of an appropriate treatment is particularly influenced by the long-term sequelae of the disease and its therapy. Thus, failure-free survival is much more important than survival alone when analyzing the long-term outcomes of children with aplastic anemia. Lack of response, relapse, and clonal evolution are problematic in the IST setting, whereas graft failure, acute and chronic graft-versus-host disease (GVHD), and infectious complications limit the success of BMT. In the present study, we compared the outcomes of children with SAA who received IST or BMT from an MFD as first-line treatment using data from nationwide IST and BMT registries. Methods PatientsBetween 1992 and 2009, a total of 599 consecutive children (younger than 17 years) with acquired SAA underwent BMT from an MFD or received IST as first-line treatment in Japan; 213 patients with an MFD underwent BMT and were registered in the Transplant Registry Unified Management Program (TRUMP) conducted by the Japanese Society for Hematopoietic Cell Transplantation, and 386 patients without an MFD were enrolled in two consecutive prospective multicenter trials (AA-92/97) conducted by the Japanese Childhood Aplastic Anemia Study Group and were initially treated with IST (Table 1). The disease severities were defined as previously reported.12,13 Underlying inherited marrow failure disorders were excluded clinically and by chromosome fragility testing. Marrow cytogenetic studies were performed for all patients, and patients with clonal cytogenetic abnormalities were excluded from this study. Patients with paroxysmal nocturnal hemoglobinuria with clinical symptoms and positive findings on the Ham test/sucrose test were also excluded from this analysis. All treatments were performed after obtaining written informed consent from patients or their parents in accordance with the Declaration of Helsinki. Immunosuppressive therapy and bone marrow transplantation proceduresThe characteristics of the treatment procedures are detailed in Table 2. Three hundred and eighty-six patients were enrolled in the AA-92 (n=84) and AA-97 (n=302) trials, and all the patients were initially treated with a combination of antithymocyte globulin and cyclosporine A. Response to IST and disease relapse were evaluated as previously reported.12 Transplantation data were collected with the use of standardized forms provided by the TRUMP. A total of 213 patients underwent BMT from an MFD as first-line treatment following the local protocols for conditioning regimens and GVHD prophylaxis. Patients who did not reach neutrophil counts >0.5×10 9 /L for 3 consecutive days after transplantation were considered to have had primary graft failure. Patients with initial engraftment in whom absolute neutrophil counts subsequently declin...

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Section: Discussionmentioning

confidence: 70%

Section: Discussionmentioning

confidence: 99%

First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy

Yoshida¹,

et al. 2014

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“…In general, patients with presenting granulocyte counts less than 0.2 x 10 9 /l tend to have a worse prognosis than others, mainly with respect to early mortality (23). In our series, 4 of the 12 patients who had granulocyte counts <0.2 x 10 9 /l responded to ATG, and another subsequently responded to cyclosporine plus prednisone.…”

Section: Discussionmentioning

confidence: 54%

Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin

deMedeiros

Bittencourt

et al. 2000

Braz J Med Biol Res

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“…In the last two decades, the survival of these patients has increased dramatically with the use of immunosupressive agents and hematopoetic stem cell transplantation (HSCT). In the pediatric age group, the data comparing the outcomes of HSCT and immunosuppressive therapy in acquired AA have shown better survival rates with HSCT [1,2]. However, comparable survival rates between HSCT and immunosuppressive therapy have also been described [3,4].…”

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confidence: 99%

The Way Forward: Aplastic Anemia and Hematopoietic Stem Cell Transplantation in the Pediatric Patient

Ünal

Uçkan

2008

Pediatric Health

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Comparison between bone marrow transplantation and antithymocyte globulin in treatment of young patients with severe aplastic anemia

Cited by 40 publications

References 27 publications

First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy

First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy

Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin

The Way Forward: Aplastic Anemia and Hematopoietic Stem Cell Transplantation in the Pediatric Patient

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