Recent advances in the diagnosis and treatment of pediatric acquired aplastic anemia

Yoshida, Nao

doi:10.1007/s12185-023-03564-4

Cited by 6 publications

(2 citation statements)

References 54 publications

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“…MSD HSCT has been the standard rst-line treatment for children with SAA/VSAA 10,17 , with nearly 90% OS 18,19 . In our retrospective study of 87 pediatric AA patients undergoing MSD peripheral blood stem cell transplantation, we noted 98% OS.…”

Section: Discussionmentioning

confidence: 99%

Early withdrawal immunosuppression improved mixed chimerism in hematopoietic stem cell transplantation for pediatric aplastic anemia

Wang,

Qin,

Luo

et al. 2024

Preprint

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Mixed chimerism (MC) occurs frequently with the risk of graft reject (GR) for aplastic anemia (AA) patients undergoing matched sibling donor (MSD) hematopoietic stem cell transplantation (HSCT). So far, no one knows how to adjust immunosuppression (IS) during MC. We retrospectively analyzed 87 consecutive pediatric patients. Early withdrawal (EW) IS and donor lymphocyte infusion (DLI) were attempted to reverse MC. The rate of MC was 26% (n = 23). Low dose cyclophosphamide (CY) (120–150 mg/kg) is an independent risk factor for MC (P = 0.0002) and increase dosage of Fludarabine (FLU) cannot eliminated it. Patients receiving 200 mg/kg CY had the lowest MC rate (8%) and best 3-year graft-versus-host disease (GVHD)/failure free survival (GFFS; 95%). Chimerism in T cells is more sensitive than that in whole blood (P = 0.001). In 17 patients with early-onset MC (ratio of DLI: 83% versus 82%), EW IS strategy is helpful to improving complete chimerism (CC) (63 vs. 295 days, P = 0.008). Our study shows that FLU is necessary to intensify CY + ATG conditioning to maintain the engraftment and 200 mg/kg CY + 150 mg/m2 FLU is recommended as a basic conditioning regimen. EW IS strategy should be considered as an important option to improve donor chimerism in early-onset MC.

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Section: Discussionmentioning

confidence: 99%

Early withdrawal immunosuppression improved mixed chimerism in hematopoietic stem cell transplantation for pediatric aplastic anemia

Wang,

Qin,

Luo

et al. 2024

Preprint

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“…During severe concurrent infections in the SAA patients, rapid immune recovery is necessary, however, selecting urgent haploidentical hematopoietic stem cell transplantation (haplo-HSCT) amidst active infection presents a significant challenge. 1,2 A previously healthy 5-year-old male with no significant past medical history, developed idiopathic hepatitis, for which he was treated with oral steroids on an outpatient basis. However, two months later, he was diagnosed with SAA (on day -55 of transplantation).…”

Section: Introductionmentioning

confidence: 99%

Successful management with urgent haploidentical-peripheral blood stem cell transplantation for a patient with severe aplastic anemia who developed disseminated fungal infection following immunosuppressive therapy

Sakaguchi,

Ikenobe,

Fujimori

et al. 2024

Preprint

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Urgent haploidentical hematopoietic cell transplantation may be considered in cases of severe aplastic anemia (SAA) without human leukocyte antigen-matched donor and suffering from severe infection. However, deciding on allogeneic transplantation in the setting of active systemic infection is challenging due to poor outcomes. This report presents a case of disseminated Magnusiomyces capitatus infection in a 5-year-old male who underwent immunosuppressive therapy for hepatitis-associated SAA. To address the critical situation, granulocyte transfusion was promptly administered from the patient’s mother, followed by unmanipulated haploidentical peripheral blood stem cell transplantation from the patient’s father with posttransplant cyclophosphamide, ultimately resulting in successful rescue.

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Successful management with urgent haploidentical‐peripheral blood stem cell transplantation for a patient with severe aplastic anaemia who developed disseminated fungal infection following immunosuppressive therapy

Ikenobe,

Fujimori,

Gocho

et al. 2024

eJHaem

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Urgent haploidentical haematopoietic cell transplantation may be considered in cases of severe aplastic anaemia (SAA) without a human leukocyte antigen‐matched donor and suffering from severe infection. However, deciding on allogeneic transplantation in the setting of active systemic infection is challenging due to poor outcomes. This report presents a case of disseminated Magnusiomyces capitatus infection in a 5‐year‐old male who underwent immunosuppressive therapy for hepatitis‐associated SAA. To address the critical situation, granulocyte transfusion was promptly administered from the patient's mother, followed by unmanipulated haploidentical peripheral blood stem cell transplantation from the patient's father with posttransplant cyclophosphamide, ultimately resulting in successful rescue.

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Recent advances in the diagnosis and treatment of pediatric acquired aplastic anemia

Cited by 6 publications

References 54 publications

Early withdrawal immunosuppression improved mixed chimerism in hematopoietic stem cell transplantation for pediatric aplastic anemia

Early withdrawal immunosuppression improved mixed chimerism in hematopoietic stem cell transplantation for pediatric aplastic anemia

Successful management with urgent haploidentical-peripheral blood stem cell transplantation for a patient with severe aplastic anemia who developed disseminated fungal infection following immunosuppressive therapy

Successful management with urgent haploidentical‐peripheral blood stem cell transplantation for a patient with severe aplastic anaemia who developed disseminated fungal infection following immunosuppressive therapy

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