Impact of CD34 Cell Dose and Conditioning Regimen on Outcomes after Haploidentical Donor Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide for Relapsed/Refractory Severe Aplastic Anemia

Arcuri, Leonardo Javier; Nabhan, Samir Kanaan; Cunha, Renato; Nichele, Samantha; Ribeiro, Andreza Alice Feitosa; Fernandes, Juliana Folloni; Daudt, Liane Esteves; Rodrigues, Ana Luiza Melo; Arrais‐Rodrigues, Celso; Seber, Adriana; Atta, Elias Hallack; Oliveira, José Salvador Rodrigues de; Funke, Vaneuza Araújo Moreira; Loth, Gisele; Darrigo, Luiz Guilherme; Paz, Alessandra Aparecida; Calixto, Rodolfo; Gomes, Alessandra Araújo; Araujo, Carlos E.; Colturato, Vergílio Antônio Rensi; Simões, Belinda Pinto; Hamerschlak, Nelson; Flowers, Mary E.D.; Pasqüini, Ricardo; Rocha, Vanderson; Bonfim, Carmem

doi:10.1016/j.bbmt.2020.09.007

Cited by 30 publications

(12 citation statements)

References 29 publications

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“…Recent literature describes use of abatacept, alpha beta (αβ) T-cell and CD19-cell depletion, and post-transplant cyclophosphamide as promising methods for GVHD prophylaxis in children with NMD. (13,(17)(18)(19) However, donor selection and the approach to GVHD prophylaxis when an HLA-matched donor is lacking, is the subject of research and until large randomized studies are available, the alternative donor selection and GVHD prophylaxis will likely remain center-specific. Only 1/5 FPBCC transplant centers performed ex-vivo T-cell depletion/CD34+ selection during the study period, and no centers performed αβ T-cell depletion, while all centers used post-transplant cyclophosphamide, which was used in the majority of haploidentical HCT recipients in this study.…”

Section: Discussionmentioning

confidence: 99%

The Impact of Graft-Versus-Host Disease on Survival in Children with Non-Malignant Disorders Receiving Allogeneic Hematopoietic Cell Transplant

Horn

Castillo

Hanif

et al. 2021

Preprint

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Background: Graft-versus-host disease (GVHD) is a common and undesirable complication of hematopoietic cell transplant (HCT) for non-malignant disorders (NMD). Understanding the incidence and risk factors for GVHD in children with NMD is an important step in developing strategies for its prevention. Study Design: This is a retrospective, registry, study that included children with NMD receiving HCT in 5 centers in Florida between 2010 and 2019. Results: Among 183 patients evaluable for GVHD, acute GVHD (aGVHD) grades I, II, III, and IV were present in 18%, 12.6%, 3.8% and 5.5% of patients, respectively. Limited and extensive chronic GVHD (cGVHD), were observed in 8.7% and 12.6% of patients. Patients with aGVHD grade III/IV had significantly lower 3-year survival rates than those without aGVHD, or those with aGVHD grade I/II (52.9% [95% confidence interval (CI) 34-83] vs. 90.1% [95% CI 84-96], vs. 98.1% [95%CI 95-100], p<0.001). Patients without cGVHD and those with limited and extensive cGVHD had 3-year survival rates of 88.9% [95%CI 84-94], 91.7% [95%CI 77-100], and 84.8% [95%CI 70-100], respectively, log rank p=0.3. Receiving transplant from an HLA-mismatched unrelated donor (MMUD), as compared to a matched related donor (MRD), increased the risk for aGVHD grade III/IV (Odds ratio 10.4 [95% CI 2.5-47.6]). There were no cases of aGVHD grade III/IV among recipients of mismatched related/haploidentical transplants. Conclusions: Grade III/IV aGVHD, which significantly reduced overall survival, was reported in 9.3% of children with NMD receiving HCT. Risk factors included HCT from a MMUD but not mismatched related donors.

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Section: Discussionmentioning

confidence: 99%

The Impact of Graft-Versus-Host Disease on Survival in Children with Non-Malignant Disorders Receiving Allogeneic Hematopoietic Cell Transplant

Horn

Castillo

Hanif

et al. 2021

Preprint

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“…A high CD34 + cell count is favorable for rapid hematopoietic recovery ( 8 ). Cell thresholds were devised to guide whether to carry out further apheresis collection.…”

Section: The Seedmentioning

confidence: 99%

“…At Peking University Institute of Hematology, the incidence of primary PGF after unmanipulated haploidentical HSCT (haplo-HSCT) was found to be approximately 5.6% ( 6 , 7 ), and sPGF developed in 5.7% of patients after allo-HSCT ( 4 ). In a prospective study, approximately 15% of patients with severe aplastic anemia (AA) who underwent haplo-SCT developed primary PGF ( 8 ). Primary PGF shows a very poor prognosis, with a 1-year overall survival (OS) rate of 25.0% ( 5 ) and 2-year OS of 6% in patients without hematopoietic recovery ( 9 ).…”

Section: Introductionmentioning

confidence: 99%

Recent Advancements in Poor Graft Function Following Hematopoietic Stem Cell Transplantation

Man

Yao

et al. 2022

Front. Immunol.

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Poor graft function (PGF) is a life-threatening complication that occurs after transplantation and has a poor prognosis. With the rapid development of haploidentical hematopoietic stem cell transplantation, the pathogenesis of PGF has become an important issue. Studies of the pathogenesis of PGF have resulted in some success in CD34+-selected stem cell boosting. Mesenchymal stem cells, N-acetyl-l-cysteine, and eltrombopag have also been investigated as therapeutic strategies for PGF. However, predicting and preventing PGF remains challenging. Here, we propose that the seed, soil, and insect theories of aplastic anemia also apply to PGF; CD34+ cells are compared to seeds; the bone marrow microenvironment to soil; and virus infection, iron overload, and donor-specific anti-human leukocyte antigen antibodies to insects. From this perspective, we summarize the available information on the common risk factors of PGF, focusing on its potential mechanism. In addition, the safety and efficacy of new strategies for treating PGF are discussed to provide a foundation for preventing and treating this complex clinical problem.

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“…Primary disease and conditioning regimen could also impact CMV infection after PTCy-haploSCT. CMV reactivation post engraftment was noted in 43.7% and 62% of transplant recipients with primary immune deficiency disorders (PIDs) (61) and relapsed/refractory SAA (62) undergoing PTCy-haploSCT, respectively. R V Raj et al then investigated the effect of conditioning intensity on the incidence of viral infection after PTCy-haploSCT (63).…”

Section: Ptcy-haplosctmentioning

confidence: 99%

CMV Infection and CMV-Specific Immune Reconstitution Following Haploidentical Stem Cell Transplantation: An Update

et al. 2021

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Haploidentical stem cell transplantation (haploSCT) has advanced to a common procedure for treating patients with hematological malignancies and immunodeficiency diseases. However, cure is seriously hampered by cytomegalovirus (CMV) infections and delayed immune reconstitution for the majority of haploidentical transplant recipients compared to HLA-matched stem cell transplantation. Three major approaches, including in vivo T-cell depletion (TCD) using antithymocyte globulin for haploSCT (in vivo TCD-haploSCT), ex vivo TCD using CD34 + positive selection for haploSCT (ex vivo TCD-haploSCT), and T-cell replete haploSCT using posttransplant cyclophosphamide (PTCy-haploSCT), are currently used worldwide. We provide an update on CMV infection and CMV-specific immune recovery in this fast-evolving field. The progress made in cellular immunotherapy of CMV infection after haploSCT is also addressed. Groundwork has been prepared for the creation of personalized avenues to enhance immune reconstitution and decrease the incidence of CMV infection after haploSCT.

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Impact of CD34 Cell Dose and Conditioning Regimen on Outcomes after Haploidentical Donor Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide for Relapsed/Refractory Severe Aplastic Anemia

Cited by 30 publications

References 29 publications

The Impact of Graft-Versus-Host Disease on Survival in Children with Non-Malignant Disorders Receiving Allogeneic Hematopoietic Cell Transplant

The Impact of Graft-Versus-Host Disease on Survival in Children with Non-Malignant Disorders Receiving Allogeneic Hematopoietic Cell Transplant

Recent Advancements in Poor Graft Function Following Hematopoietic Stem Cell Transplantation

CMV Infection and CMV-Specific Immune Reconstitution Following Haploidentical Stem Cell Transplantation: An Update

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