Haploidentical hematopoietic stem cell transplantation for nonresponders to immunosuppressive therapy against acquired severe aplastic anemia

Liu, L; Wang, X; Jin, Song; Hao, Lin; Y, Zhang; Zhang, X; Wu, Depei

doi:10.1038/bmt.2015.249

Cited by 12 publications

(8 citation statements)

References 11 publications

(15 reference statements)

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“…Although the salvage HSCT after IST failure group achieved acceptable OS and FFS, the rates of aGVHD and cGVHD were higher in the salvage HSCT group compared with the frontline MSD-HSCT group, the OS of patients age >12 years decreased with increasing age, and the risk of secondary tumors increased [9,16]. In a study of patients who received haplo-HSCT after IST failure, the engraftment rate was 96.15%; the rates of aGVHD, grade III-IV aGVHD, and cGVHD were 61.52 § 9.5%, 15.55 § 8.48%, and 81.07 § 11.51%, respectively; and 3-year OS and FFS were 76.30 § 9.70% and 76.30 § 9.70%, respectively [17]. In our study, 8 patients underwent haplo-HSCT and 4 patients underwent MUD-HSCT in the salvage HSCT group; the engraftment rate at day +30 was 91.7%; the rates of aGVHD, grade II-IV aGVHD, and cGVHD were 66.7%, 25.0%, and 33.3%, respectively; and EFS was 35.0 § 15.4%.…”

Section: Discussionmentioning

confidence: 99%

Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor

Yang

Yuan

et al. 2019

Biology of Blood and Marrow Transplantation

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A B S T R A C TWe compared the outcomes of immunosuppressive therapy (IST) with those of T cell-replete haploidentical donor hematopoietic stem cell transplantation (haplo-HSCT) in children and adolescents with severe aplastic anemia (SAA). The medical records of 49 patients with SAA who received frontline IST (n = 29) or frontline haplo-HSCT (n = 20) between 2012 and 2016 were analyzed retrospectively. Fourteen patients responded after the first IST, and 1 patient responded after the second IST in the frontline IST group; 12 patients underwent salvage HSCT after IST failure. Sixteen of the 20 patients who underwent frontline haplo-HSCT survived without treatment failure. The 3-year overall survival of the frontline IST group was comparable to that of the frontline haplo-HSCT group (79.3 § 7.5% versus 85.0 § 8.0%; x 2 = 0.110; P = .740). The 3-year failure-free survival was lower in the frontline IST group compared with the frontline haplo-HSCT group (35.9 § 10.9% versus 80.0 § 8.9%; x 2 = 4.089; P = .043). Five patients of the IST group who underwent salvage HSCT achieved long survival without event. The event-free survival was lower in the salvage HSCT group compared with the haplo-HSCT group (41.7 § 14.2% versus 80.0 § 8.9%; x 2 = 3.992; P = .046), and the incidences of acute GVHD, grade II-IV acute GVHD, chronic GVHD, and severe infection were comparable between the 2 groups. Our results suggest that frontline haplo-HSCT may be a better treatment than IST for children and adolescents with SAA who lack an HLA age-matched familial donor.

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Section: Discussionmentioning

confidence: 99%

Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor

Yang

Yuan

et al. 2019

Biology of Blood and Marrow Transplantation

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“…As a result, 32 records were excluded, and 29 studies met the inclusion criteria. The included studies were divided into two groups (MSCs [31][32][33][34][35][36][37][38]; no MSCs [22,24,25,[39][40][41][42][43][44][45][46][47][48][49][50][51][52][53][54][55][56]) based on whether they applied the MSCs or not. The selection process is illustrated in Fig.…”

Section: Search Resultsmentioning

confidence: 99%

Mesenchymal stromal cells as prophylaxis for graft-versus-host disease in haplo-identical hematopoietic stem cell transplantation recipients with severe aplastic anemia?—a systematic review and meta-analysis

Zhao

et al. 2021

Stem Cell Res Ther

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Background Mesenchymal stromal cells (MSCs) are an emerging prophylaxis option for graft-versus-host disease (GVHD) in haplo-identical hematopoietic stem cell transplantation (haplo-HSCT) recipients with severe aplastic anemia (SAA), but studies have reported inconsistent results. This systematic review and meta-analysis evaluates the efficacy of MSCs as prophylaxis for GVHD in SAA patients with haplo-HSCT. Methods Studies were retrieved from PubMed, EMBASE, Cochrane, Web of Science, and http://clinicaltrials.gov from establishment to February 2020. Twenty-nine single-arm studies (n = 1456) were included, in which eight (n = 241) studies combined with MSCs and eleven (n = 1215) reports without MSCs in haplo-HSCT for SAA patients. The primary outcomes were the incidences of GVHD. Other outcomes included 2-year overall survival (OS) and the incidence of cytomegalovirus (CMV) infection. Odds ratios (ORs) were calculated to compare the results pooled through random or fixed effects models. Results Between MSCs and no MSCs groups, no significant differences were found in the pooled incidences of acute GVHD (56.0%, 95% CI 48.6–63.5% vs. 47.2%, 95% CI 29.0–65.4%; OR 1.43, 95% CI 0.91–2.25; p = 0.123), grade II–IV acute GVHD (29.8%, 95% CI 24.1–35.5% vs. 30.6%, 95% CI 26.6–34.6%; OR 0.97, 95% CI 0.70–1.32; p = 0.889), and chronic GVHD (25.4%, 95% CI 19.8–31.0% vs. 30.0%, 95% CI 23.3–36.6%; OR 0.79, 95% CI 0.56–1.11; p = 0.187). Furtherly, there was no obvious difference in 2-year OS (OR 0.98, 95% CI 0.60–1.61; p = 1.000) and incidence of CMV infection (OR 0.61, 95% CI 0.40–1.92; p = 0.018). Conclusions Our meta-analysis indicates that the prophylactic use of MSC co-transplantation is not an effective option for SAA patients undergoing haplo-HSCT. Hence, the general co-transplantation of MSCs for SAA haplo-HSCT recipients may lack evidence-based practice.

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“…As a result, 32 records were excluded, and 29 studies met the inclusion criteria. The included studies were divided into two groups (MSCs [31][32][33][34][35][36][37][38] ; no MSCs 22,25,[39][40][41][42][43][44][45][46][47][48][49][50][51][52][53][54][55][56][57] ) based on whether they applied the MSCs or not. The selection process is illustrated in Fig.…”

Section: Search Resultsmentioning

confidence: 99%

Mesenchymal Stromal Cells as Prophylaxis for Graft-versus-host Disease in Haplo-identical Hematopoietic Stem Cell Transplantation Recipients With Severe Aplastic Anemia? - A Systematic Review and Meta-analysis

Zhao

et al. 2020

Preprint

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Background: Mesenchymal stromal cells (MSCs) are an emerging prophylaxis option for graft-versus-host disease (GVHD) in haplo-identical hematopoietic stem cell transplantation (haplo-HSCT) recipients with severe aplastic anemia (SAA), but studies have reported inconsistent results. This systematic review and meta-analysis evaluates the efficacy of MSCs as prophylaxis for GVHD in SAA patients with haplo-HSCT. Methods: Studies were retrieved from PubMed, EMBASE, Cochrane, Web of Science and http://clinicaltrials.gov from establishment to February 2020. Twenty-nine single-arm studies (n = 1456) were included, eight (n = 241) studies combined with MSCs and eleven (n = 1215) reports without MSCs in haplo-HSCT for SAA patients. The primary outcomes were the incidences of GVHD. Other outcomes included 2-year overall survival (OS) and the prevalence of cytomegalovirus viremia (CMV). Odds ratios (ORs) were calculated to compare the results pooled through random or fixed effects models. Results: Between MSCs and no MSCs groups, no significant differences were found in the pooled incidences of acute GVHD (56.0%, 95%CI: 48.6%-63.5% vs. 47.2%, 95%CI: 29.0%-65.4%; OR 1.43, 95%CI: 0.91-2.25; p = 0.123), grade II-IV acute GVHD (29.8%, 95%CI: 24.1%-35.5% vs. 30.6%, 95%CI: 26.6%-34.6%; OR 0.97, 95%CI: 0.70-1.32; p = 0.889), chronic GVHD (25.4%, 95%CI: 19.8%-31.0% vs. 30.0%, 95%CI, 23.3%-36.6%; OR 0.79, 95%CI 0.56-1.11; p = 0.187). Furtherly, there was no obvious differences in 2-year OS (OR 0.98, 95%CI 0.60-1.61; p = 1.000) and prevalence of CMV (OR 0.61, 95%CI 0.40-1.92; p = 0.018).Conclusions: Our meta-analysis indicates that the prophylactic use of MSCs co-transplantation is not an effective option for SAA patients undergoing haplo-HSCT. Hence, the general co-transplantation of MSCs for SAA haplo-HSCT recipients may lack of evidence-based practice.

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Haploidentical hematopoietic stem cell transplantation for nonresponders to immunosuppressive therapy against acquired severe aplastic anemia

Cited by 12 publications

References 11 publications

Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor

Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor

Mesenchymal stromal cells as prophylaxis for graft-versus-host disease in haplo-identical hematopoietic stem cell transplantation recipients with severe aplastic anemia?—a systematic review and meta-analysis

Mesenchymal Stromal Cells as Prophylaxis for Graft-versus-host Disease in Haplo-identical Hematopoietic Stem Cell Transplantation Recipients With Severe Aplastic Anemia? - A Systematic Review and Meta-analysis

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