Improved outcome of patients older than 30 years receiving HLA-identical sibling hematopoietic stem cell transplantation for severe acquired aplastic anemia using fludarabine-based conditioning: a comparison with conventional conditioning regimen

Maury, Sébastien; Bacigalupo, Andrea; Anderlini, Paolo; Aljurf, Mahmoud; Marsh, Judith; Socié, Gérard; Oneto, Rosi; Passweg, Jakob

doi:10.3324/haematol.2009.006916

Cited by 111 publications

(82 citation statements)

References 20 publications

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“…The OS was also better. 15 In a phase II prospective study, no GF was found in 28 patients with SAA who received Flu/CY/ATG conditioning regimen. 16 BU is an alkylating agent with low immunosuppressive activity.…”

Section: Discussionmentioning

confidence: 99%

Good outcome of haploidentical hematopoietic SCT as a salvage therapy in children and adolescents with acquired severe aplastic anemia

Wang

Zheng

Yan

et al. 2014

Bone Marrow Transplant

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Haploidentical hematopoietic SCT (haplo-HSCT) is to be established in patients with acquired severe aplastic anemia (SAA) refractory to immunosuppressive therapy and lacking HLA-matched related or unrelated donors. Graft failure (GF) and GVHD have been major obstacles to HSCT. A total of 17 children and adolescents with SAA underwent haplo-HSCT in our center. The conditioning regimen consisted of BU, fludarabine, CY and anti-thymocyte globulin. All patients received cyclosporine, short-term MTX, mycophenolate mofetil and basiliximab for GVHD prophylaxis. Mesenchymal stem cells derived from unrelated umbilical cord were infused on day 1. Neutrophil engraftment was achieved in all 17 patients in a median time of 16 days (range 9-25 days). The median time of platelet engraftment was 22 days (range 9-95 days) in 16 patients. The cumulative incidence (CI) of II-IV acute GVHD (aGVHD) at day +100 was 30.53 ± 11.12% and III-IV aGVHD occurred in only one patient. The CI of chronic GVHD was 21.25 ± 13.31%. Secondary GF with autologous hematopoiesis recovery occurred in one patient. The OS was 71.60 ± 17.00% at a median follow-up of 362 (36-1321) days. These limited promising data suggest that haplo-HSCT is feasible as a salvage therapy for children and adolescents with refractory SAA who lack matched donors.

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Section: Discussionmentioning

confidence: 99%

Good outcome of haploidentical hematopoietic SCT as a salvage therapy in children and adolescents with acquired severe aplastic anemia

Wang

Zheng

Yan

et al. 2014

Bone Marrow Transplant

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“…We, therefore, suggest that UD allo-HSCT should be safely considered up to 30 years of age, approaching the clinically relevant age cut off of 40 years in the setting of HLA-identical sibling allo-HSCT. 22,23 For older patients, UD allo-HSCT should be performed with caution in highly selected patients, especially in the presence of additional poor risk factors such as comorbidities. 5 Regarding the timing of allo-HSCT, we found better OS when allo-HSCT was performed earlier, within the first year following diagnosis.…”

Section: Discussionmentioning

confidence: 99%

Unrelated alternative donor transplantation for severe acquired aplastic anemia: a study from the French Society of Bone Marrow Transplantation and Cell Therapies and the EBMT Severe Aplastic Anemia Working Party

et al. 2016

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U nrelated allogeneic transplantation for severe aplastic anemia is a treatment option after immunosuppressive treatment failure in the absence of a matched sibling donor. Age, delay between disease diagnosis and transplantation, and HLA matching are the key factors in transplantation decisions, but their combined impact on patient outcomes remains unclear. Using the French Society of Bone Marrow Transplantation and Cell Therapies registry, we analyzed all consecutive patients (n=139) who underwent a first allogeneic transplantation for idiopathic severe aplastic anemia from an unrelated donor between 2000 and 2012. In an adjusted multivariate model, age over 30 years (Hazard Ratio=2.39; P=0.011), time from diagnosis to transplantation over 12 months (Hazard Ratio=2.18; P=0.027) and the use of a 9/10 mismatched unrelated donor (Hazard Ratio=2.14; P=0.036) were independent risk factors that significantly worsened overall survival. Accordingly, we built a predictive score using these three parameters, considering patients at low (zero or one risk factors, n=94) or high (two or three risk factors, n=45) risk. High-risk patients had significantly shorter survival (Hazard Ratio=3.04; P<0.001). The score was then confirmed on an independent cohort from the European Group for Blood and Marrow Transplantation database of 296 patients, with shorter survival in patients with at least 2 risk factors (Hazard Ratio=2.13; P=0.005) In conclusion, a simple score using age, transplantation timing and HLA matching would appear useful to help physicians in the daily care of patients with severe aplastic anemia.

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“…Recently, Flu-based preconditioning regimen is widely used in HSCT for SAA from MSD or alternative donors. [14][15][16] In such situations, low-dose TBI is usually combined with chemotherapeutic agents to avoid rejection in Japan. Adverse effects of radiation include not only LM but also endocrinological disorders, such as hypothyroidism or hypogonadism, or other organ dysfunctions.…”

Section: Discussionmentioning

confidence: 99%

Long-term outcome of childhood aplastic anemia patients who underwent allogeneic hematopoietic SCT from an HLA-matched sibling donor in Japan

Kikuchi

Yabe

Katô³

et al. 2012

Bone Marrow Transplant

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We report long-term outcomes of 329 childhood severe aplastic anemia (SAA) patients who underwent hematopoietic SCT (HSCT) from an HLA-matched sibling donor in the Japanese Hematopoietic Cell Transplantation Registry. OS and EFS at 10 years were as high as 89.7 þ / À 1.7% and 85.5 þ / À 2.0%, respectively. Five cases of late malignancies (LM) were identified (malignant peripheral nerve sheath tumor, thyroid carcinoma, colon carcinoma, MDS and hepatoblastoma). Cumulative incidence of LM was 0.8% at 10 years and 2.5% at 20 years, respectively, which was lower than that in previous reports. This low incidence is in keeping with the low occurrence of skin cancer in Japanese population and of acute GVHD in our study group. Radiation-containing conditioning was not significantly associated with the incidence of LM after HSCT probably because of absolute low patient number who developed LM in our series. In terms of LM development after HSCT, low-dose TBI in HSCT for SAA to avoid graft rejection, which is commonly used in Japan, might be tolerable in the Japanese population because of its low incidence. Keywords: aplastic anemia; childhood; hematopoietic SCT; long-term outcome; late malignancies INTRODUCTION Aplastic anemia is a hematopoietic disorder characterized by pancytopenia and BM hypoplasia. Therapeutic options for severe aplastic anemia (SAA) are immunosuppressive therapy and hematopoietic SCT (HSCT). In children, if an HLA-matched sibling donor (MSD) is available, HSCT is the first line of therapy for SAA. HSCT from MSD is an established standard therapy for SAA in children and high survival rates have already been reported. However, little is known about long-term outcomes of these children.Late malignancies (LM) are serious complications for patients who undergo HSCT. 3,4 In SAA, LM are also reported in the patients with HSCT and its occurrence is related to radiation-containing conditioning regimen and GVHD grade in adults, 5,6 however, the incidence and prognostic factors of LM in children with SAA who undergo HSCT are totally unknown.In this article, we report long-term outcomes of childhood SAA patients who underwent HSCT from MSD in the centers of the Japanese Hematopoietic Cell Transplantation Registry and describe the occurrence of LM.

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Improved outcome of patients older than 30 years receiving HLA-identical sibling hematopoietic stem cell transplantation for severe acquired aplastic anemia using fludarabine-based conditioning: a comparison with conventional conditioning regimen

Cited by 111 publications

References 20 publications

Good outcome of haploidentical hematopoietic SCT as a salvage therapy in children and adolescents with acquired severe aplastic anemia

Good outcome of haploidentical hematopoietic SCT as a salvage therapy in children and adolescents with acquired severe aplastic anemia

Unrelated alternative donor transplantation for severe acquired aplastic anemia: a study from the French Society of Bone Marrow Transplantation and Cell Therapies and the EBMT Severe Aplastic Anemia Working Party

Long-term outcome of childhood aplastic anemia patients who underwent allogeneic hematopoietic SCT from an HLA-matched sibling donor in Japan

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