Predictive Factors in Chronic Graft-Versus-Host Disease in Patients with Aplastic Anemia Treated by Marrow Transplantation from HLA-Identical Siblings

Storb, Rainer; Prentice, Ross L.; Sullivan, Keith M.; Shulman, Howard M.; Deeg, H. Joachim; Doney, Kristine; Cd, Buckner; Clift, Reginald A.; Witherspoon, Robert P.; Appelbaum, F; Sanders, Jean E.; Stewart, Patricia; Thomas, E. Donnall

doi:10.7326/0003-4819-98-4-461

Cited by 274 publications

(150 citation statements)

References 31 publications

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“…19,20 Children surviving more than 14 days and 100 days post transplantation and with evidence of donor engraftment were evaluated for the occurrence of aGVHD and cGVHD, respectively. Relapse was defined on the basis of morphological evidence of leukemia in BM, or at other extramedullary sites.…”

Section: Definitionsmentioning

confidence: 99%

Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneic hematopoietic cell transplantation in the aieop AML-2002/01 study.

Locatelli

Masetti

Rondelli

et al. 2014

Bone Marrow Transplant

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on behalf of AIEOP BMT Working GroupWe analyzed the outcome of 243 children with high-risk (HR) AML in first CR1 enrolled in the AIEOP-2002/01 protocol, who were given either allogeneic (ALLO; n = 141) or autologous (AUTO; n = 102) hematopoietic SCT (HSCT), depending on the availability of a HLA-compatible sibling. Infants, patients with AML-M7, or complex karyotype or those with FLT3-ITD, were eligible to be transplanted also from alternative donors. All patients received a myeloablative regimen combining BU, Cyclophosphamide and Melphalan; AUTO-HSCT patients received BM cells in most cases, while in children given ALLO-HSCT stem cell source was BM in 96, peripheral blood in 19 and cord blood in 26. With a median follow-up of 57 months (range 12-130), the probability of disease-free survival (DFS) was 73% and 63% in patients given either ALLO-or AUTO-HSCT, respectively (P = NS). Although the cumulative incidence (CI) of relapse was lower in ALLO-than in AUTO-HSCT recipients (17% vs 28%, respectively; P = 0.043), the CI of TRM was 7% in both groups. Patients transplanted with unrelated donor cord blood had a remarkable 92.3% 8-year DFS probability. Altogether, these data confirm that HSCT is a suitable option for preventing leukemia recurrence in HR children with CR1 AML.

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Section: Definitionsmentioning

confidence: 99%

Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneic hematopoietic cell transplantation in the aieop AML-2002/01 study.

Locatelli

Masetti

Rondelli

et al. 2014

Bone Marrow Transplant

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“…A classical study from Storb et al 19 registered an increased incidence of cGVHD associated with an increment in the number of lymphoid cells in patients with aplastic anemia. In this study, multiply transfused patients where given unirradiated buffy-coat cells from the marrow donor, in addition to bone marrow, in order to increase the number of stem cells, and provide donor lymphoid cells, which may be beneficial in enhancing hematopoietic engraftment.…”

Section: Discussionmentioning

confidence: 99%

“…For matching, every patient in the allo-BMT group shared the following characteristics with the corresponding patient in the allo-PBT group: [18][19][20] age (interval of ±5 years), grade of previous acute GVHD, male patient with female donor (whether or not she had borne children), and phase of disease. Control patient characteristics are listed in Table 1.…”

Section: Historical Control Groupmentioning

confidence: 99%

Chronic graft-versus-host disease after allogeneic peripheral blood progenitor cell or bone marrow transplantation from matched related donors. A case-control study

Solano

Martı́nez

Brunet

et al. 1998

Bone Marrow Transplant

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Summary:We retrospectively compared the incidence and clinical characteristics of cGVHD in 37 allo-PBT recipients transplanted between July 1994 and October 1996 and 37 historical control allo-BMT recipients in a casecontrol study. All patients received a first unmanipulated transplant, graft from an HLA-identical sibling donor, with CsA-MTX GVHD prophylaxis and survived more than 100 days after transplant. PBT and BMT groups were well matched for age, grade of acute GVHD, male patients grafted from female donors, and phase of disease. The median CD34 + and CD3 + cell numbers infused in the PBT group were 5.2 × 10 6 /kg and 307 × 10 6 /kg, respectively. The median time to an ANC greater than 0.5 × 10 9 /l was 16 days (range 11-22) after PBT and 22 days (range 14-36) after BMT (P Ͻ 0.001). The median time to a platelet count greater than 20 × 10 9 /l was 15 days (range 6-43) after PBT and 28 days (range 12-68) after BMT (P Ͻ 0.001). Median follow-up was 12.3 months (range 5.4-30.3) and 58.7 months (range 4-122.3), for patients receiving PBT and BMT, respectively. Seventeen out of 37 (46%) PBT recipients, vs nine out of 37 (24%) BM recipients developed cGVHD. Actuarial probability of cGVHD at 1 year was 59% (95% CI, 39-79) in the PBT group vs 27% (95% CI, 12-42) in the BM group (P = 0.01). Cumulative incidence estimate of cGVHD was 51% and 25%, for patients receiving PBT and BMT respectively (P = 0.03). Clinical characteristics of cGVHD and response to therapy were similar in both groups, except for a higher incidence of de novo cGVHD in the PBT group. Our results suggest that as compared with BMT, PBT may result in an increased incidence of cGVHD.

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“…Children alive at day þ 100 post-transplantation with sustained donor engraftment were considered to be evaluable for chronic GVHD, which was classified according to previously reported criteria. 15 Treatment of GVHD was administered according to protocols in use at each single institution. Myeloid engraftment was defined as the first of three consecutive days with a neutrophil count 40.5 Â 10 9 /l, and platelet engraftment as the first of three consecutive days with an unsupported platelet count 420 Â 10 9 /l.…”

Section: Patient Characteristicsmentioning

confidence: 99%

Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study

et al. 2011

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We report on the outcome of children with advanced primary myelodysplastic syndrome (MDS) transplanted from an HLAmatched sibling (MSD) or an unrelated donor (UD) following a preparative regimen with busulfan, cyclophosphamide and melphalan. Ninety-seven patients with refractory anemia with excess blasts (RAEB, n ¼ 53), RAEB in transformation (RAEB-T, n ¼ 29) and myelodysplasia-related acute myeloid leukemia (MDR-AML, n ¼ 15) enrolled in the European Working Group of MDS in Childhood (EWOG-MDS) 98 study and given hematopoietic stem cell transplantation (HSCT) were analyzed. Median age at HSCT was 11.1 years (range 1.4-19.0). Thirty-nine children were transplanted from an MSD, whereas 58 were given the allograft from a UD (n ¼ 57) or alternative family donor (n ¼ 1). Stem cell source was bone marrow (n ¼ 69) or peripheral blood (n ¼ 28). With a median follow-up of 3.9 years (range 0.1-10.9), the 5-year probability of overall survival is 63%, while the 5-year cumulative incidence of transplantation-related mortality (TRM) and relapse is 21% each. Age at HSCT greater than 12 years, interval between diagnosis and HSCT longer than 4 months, and occurrence of acute or extensive chronic graft-versus-host disease were associated with increased TRM. The risk of relapse increased with more advanced disease. This study indicates that HSCT following a myeloablative preparative regimen offers a high probability of survival for children with advanced MDS.

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Predictive Factors in Chronic Graft-Versus-Host Disease in Patients with Aplastic Anemia Treated by Marrow Transplantation from HLA-Identical Siblings

Cited by 274 publications

References 31 publications

Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneic hematopoietic cell transplantation in the aieop AML-2002/01 study.

Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneic hematopoietic cell transplantation in the aieop AML-2002/01 study.

Chronic graft-versus-host disease after allogeneic peripheral blood progenitor cell or bone marrow transplantation from matched related donors. A case-control study

Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study

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