Treatment of acquired aplastic anemia in children

Kojima, Seiji

doi:10.1179/102453312x13336169155051

Cited by 5 publications

(12 citation statements)

References 17 publications

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“…Haplo‐HSCT has been used as a third‐line treatment option for childhood SAA/VSAA patients without an MSD or MUD and failing one or two courses of IST (Kojima, ; Scheinberg & Young, ; Hartung et al , ; Shin et al , ). In recent years, the outcome of haplo‐HSCT has improved remarkably due to superior methods for diagnosis and differential diagnosis, high resolution HLA typing methods, new drugs for conditioning (fludarabine, alemtuzumab), better supportive treatment and cell therapy strategies, such as partial T‐cell depletion, purified CD34 cells and co‐transplantation of mesenchymal stem cells(Ciceri et al , ; Hartung et al , ; DeZern & Guinan, ).…”

Section: Discussionmentioning

confidence: 99%

“…Therefore, repeat IST should not be the preferential selection for those patients without a MUD who fail first course IST and. Some experts have proposed the upfront haplo‐HSCT for childhood and young adults with SAA/VSAA who do not have a MSD or MUD, especially in the refractory and emergency settings, due to the promising outcomes and easy accessibility of donors (Kojima, ; Chen et al , ; Marsh & Kulasekararaj, ).…”

Section: Discussionmentioning

confidence: 99%

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Unmanipulated haploidentical haematopoietic stem cell transplantation for children with severe aplastic anaemia

et al. 2016

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Haploidentical haematopoietic stem cell transplantation (haplo-HSCT) used to be a third-line treatment option for childhood severe aplastic anaemia (SAA). We conducted this retrospective study of 36 children (38 transplants) who received haplo-HSCT from human leucocyte antigen (HLA)-mismatched related donors between July 2002 and November 2013 at five HSCT centres in China, including 17 cases that were 5/6 HLA matched (Group 1) and 21 that were 4/6 or 3/6 HLA matched (Group 2). Although patients in Group 2 had a higher incidence of grade II-IV acute graft-versus-host disease (57·9% vs. 5·9%, P = 0·001), they had similar rates of graft failure (5·3% vs. 5·9%, P = 0·742) and overall survival (80·8% vs. 93·8%, P = 0·234) as Group 1. Unmanipulated haplo-HSCT is an effective treatment for SAA children with satisfactory outcome of this cohort, especially in the 5/6 HLA-matched group. For patients in critical situations, such as unresponsive to immunosuppressive therapy, refractory infection and failing first HSCT, to bring forward the timing of haplo-HSCT is a feasible salvage strategy with better and faster donor accessibility.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Unmanipulated haploidentical haematopoietic stem cell transplantation for children with severe aplastic anaemia

et al. 2016

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“…What is more, two multicenter studies from China indicated that haploidentical transplantation applying unified regimen, as salvage or upfront choice, both achieved comparable outcomes with MRD SCT . Due to easy availability of donor and remarkable improvement in transplant technology, transplantation from a haploidentical donor should perhaps be considered more than a salvage option in children . To evaluate the first‐line treatment decision in childhood SAA, we retrospectively compared the long‐term outcome of all children with severe AA treated between 2007 and 2016 at our center.…”

Section: Introductionmentioning

confidence: 99%

First‐line choice for severe aplastic anemia in children: Transplantation from a haploidentical donor vs immunosuppressive therapy

Cheng

Zhang

et al. 2018

Clinical Transplantation

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We retrospectively compared the outcomes of children with severe aplastic anemia (SAA) who received immunosuppressive therapy (IST) or who underwent hematopoietic stem cell transplantation (HSCT) from a haploidentical donor (HID), between 2007 and 2016. A total of 52 children with SAA under the age of 17 years were initially treated with IST (n = 24) or haploidentical HSCT (n = 28) as first-line treatment. The estimated 10-year overall survival was 73.4 ± 12.6% and 89.3 ± 5.8% in patients treated with IST or HID-HSCT (P = .806). The failure-free survival was significantly inferior in patients receiving IST than in those undergoing transplantation from an HID (52.6 ± 10.5% vs 89.3 ± 5.8, P = .008). In univariate and multivariate analysis, the choice of first-line immunosuppressive therapy was the only adverse predictor for failure-free survival. At the last follow-up, completely normal blood count was observed in 11 of 20 (55.0%) and 24 of 25 (96.0%) live cases in IST and HID-HSCT cohort (P = .003). These suggest that HSCT from a haploidentical donor could be considered as first-line treatment in children who lack a matched related donor, especially in experienced transplantation centers.

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“…Multiple post‐HSCT trials and studies have indicated OS and EFS rates fluctuating from 75% to 100% . Despite these persuasive reports, roughly 30% of the SAA candidates possess an MRD .…”

mentioning

confidence: 99%

Matched related donor hematopoietic stem cell transplantation results in a long‐term follow‐up of a pediatric acquired severe aplastic anemia subset: A stem cell source perspective

Hamidieh

Mozafari

Noshad

et al. 2015

Pediatric Transplantation

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HSCT has substantially improved pediatric acquired SAA patients' outcomes. Retrospectively, we attempted to assess the outcome of MRD HSCT in 65 pediatric patients referred to a single center from 1992 to 2012. We were particularly interested to find out whether source of SC (PB, n = 40 and BM, n = 25) significantly impacts EFS and GVHD incidence. With a median follow-up of 45 months, total EFS was 87.7%; EFS for PB and BM groups was 87.5% and 88%, respectively. Acute GVHD (grades 3-4) occurred in 13 patients (PB, n = 10 [25%] and BM, n = 3 [12%]), acute GVHD (grades 2-4) occurred in 24 (PB, n = 16 [40%] and BM, n = 8 [32%]). Extensive chronic GVHD occurred in five patients (PB, n = 3 [7.5%] and BM, n = 2 [8%]). Cox regression revealed that elapsed time of <10 months between diagnosis and HSCT is associated with improved survival (hazard ratio, 95% CI = 1.204, 1.010-1.434, p = 0.038). SC source did not significantly affect EFS, incidence of acute GVHD (grades 3-4), or extensive chronic GVHD (p = 0.938, 0.121, and 0.487, respectively). Based on our findings, pediatric acquired SAA patients are benefitted most if MRD-HSCT is carried out early in disease process and SC source does not affect outcome of MRD-HSCT in these patients.

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Treatment of acquired aplastic anemia in children

Cited by 5 publications

References 17 publications

Unmanipulated haploidentical haematopoietic stem cell transplantation for children with severe aplastic anaemia

Unmanipulated haploidentical haematopoietic stem cell transplantation for children with severe aplastic anaemia

First‐line choice for severe aplastic anemia in children: Transplantation from a haploidentical donor vs immunosuppressive therapy

Matched related donor hematopoietic stem cell transplantation results in a long‐term follow‐up of a pediatric acquired severe aplastic anemia subset: A stem cell source perspective

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