Successful long‐term hematological and immunological reconstitution by autologous cord blood transplantation combined with post‐transplant immunosuppression in two children with severe aplastic anemia

Georgia, Avgerinou; Oikonomopoulou, Christina; Kaisari, Aikaterini; Ioannidou, Elda; Komitopoulou, Anna; Paisiou, Anna; Tourkantoni, Natalia; Maria, Filippidou; Kattamis, Antonios; Vessalas, George; Peristeri, Ioulia; Goussetis, Evgenios; Kitra, Vasiliki

doi:10.1111/petr.13320

Cited by 5 publications

(1 citation statement)

References 17 publications

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“…It is of note, however, that at +30 days after transplantation, the engrafted HSCs were derived from CB in 5 of the 109 patients. In unique cases, a successful autologous CB transplantation was reported in SAA, but there were differences regarding the use of conditioning therapy before the transplantation (229)(230)(231)(232).…”

Section: Cord Blood Transplantationsmentioning

confidence: 99%

The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults

Piekarska,

Pawelec,

Szmigielska-Kapłon

et al. 2024

Front. Immunol.

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Acquired aplastic anemia (AA) is an immune-mediated bone marrow (BM) failure where marrow disruption is driven by a cytotoxic T-cell–mediated autoimmune attack against hematopoietic stem cells. The key diagnostic challenge in children, but also in adults, is to exclude the possible underlying congenital condition and myelodysplasia. The choice of treatment options, either allogeneic hematopoietic cell transplantation (alloHCT) or immunosuppressive therapy (IST), depends on the patient’s age, comorbidities, and access to a suitable donor and effective therapeutic agents. Since 2022, horse antithymocyte globulin (hATG) has been available again in Europe and is recommended for IST as a more effective option than rabbit ATG. Therefore, an update on immunosuppressive strategies is warranted. Despite an improved response to the new immunosuppression protocols with hATG and eltrombopag, some patients are not cured or remain at risk of aplasia relapse or clonal evolution and require postponed alloHCT. The transplantation field has evolved, becoming safer and more accessible. Upfront alloHCT from unrelated donors is becoming a tempting option. With the use of posttransplant cyclophosphamide, haploidentical HCT offers promising outcomes also in AA. In this paper, we present the state of the art in the management of severe AA for pediatric and adult patients based on the available guidelines and recently published studies.

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Section: Cord Blood Transplantationsmentioning

confidence: 99%

The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults

Piekarska,

Pawelec,

Szmigielska-Kapłon

et al. 2024

Front. Immunol.

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A multicentre trial of intensive immunosuppressive therapy combined with umbilical cord blood for the treatment of severe aplastic anaemia

Zhou

Zhang

et al. 2022

Ann Hematol

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Immunosuppressive therapy (IST) is an effective treatment regimen for severe aplastic anaemia (SAA) patients without HLA-identical donors. This study further compared the outcomes between IST and IIST-UCB in SAA on the basis of research shown that IST combined with umbilical cord blood infusion (IIST-UCB) treated effectively. A total of 123 patients from 11 hospitals in China were enrolled. Sixty-nine patients in IIST-UCB group were treated with ATG + CsA + CTX combined with cord blood, while 54 patients in IST group with ATG + CsA. The overall remission rates (ORRs), complete remission (CR) rates and partial response (PR) rates of IIST-UCB group and IST group at 3 months were 69.67% vs 51.85% (P = .045), 21.74% vs 3.7% (P = .004) and 47.83% vs 48.15% (P = .972), respectively. After 6 months of treatment, they were 76.81% vs 57.41% (P = .022), 37.68% vs 11.11% (P = .001) and 39.13% vs 46.30% (P = .425), respectively. After 1 year of treatment, they were 85.51% vs 61.11% (P = .002), 59.42% vs 25.93% (P = .000) and 26.09% vs 35.19% (P = .275), respectively. The ORRs and CR rates of IIST-UCB group were both significantly higher than IST group after 3 months, 6 months and 1 year of treatment. The neutrophil granulocyte, platelet and haemoglobin recovery times of IIST-UCB group were significantly shorter than IST group. Compared with standard IST, IIST-UCB as an effective therapy for SAA patients without HLA-identical donors accelerated the haematopoietic reconstitution, resulting in higher early CR rates.

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Successful Bone Marrow Recovery After an Immunoablative Regimen With Autologous Cord Blood Transplant in a Child With Idiopathic Severe Aplastic Anemia: A Case Report

Rosa

Gajek

Salamonowicz

et al. 2020

Transplantation Proceedings

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Successful long‐term hematological and immunological reconstitution by autologous cord blood transplantation combined with post‐transplant immunosuppression in two children with severe aplastic anemia

Cited by 5 publications

References 17 publications

The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults

The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults

A multicentre trial of intensive immunosuppressive therapy combined with umbilical cord blood for the treatment of severe aplastic anaemia

Successful Bone Marrow Recovery After an Immunoablative Regimen With Autologous Cord Blood Transplant in a Child With Idiopathic Severe Aplastic Anemia: A Case Report

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