Haploidentical Stem Cell Transplantation After TCR-αβ+ and CD19+ Cells Depletion In Children With Congenital Non-Malignant Disease

Giardino, Stefano; Bagnasco, Francesca; Falco, Michela; Miano, Maurizio; Pierri, Filomena; Risso, Marco; Terranova, Paola; Martino, Daniela Di; Massaccesi, Erika; Ricci, Marianna; Chianucci, Benedetta; Dell’Orso, Gianluca; Sabatini, Federica; Podestà, Marina; Lanino, Edoardo; Faraci, Maura

doi:10.1016/j.jtct.2022.04.002

Cited by 13 publications

(15 citation statements)

References 45 publications

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“…While graft failure was low in patients receiving αßTB depletion for malignant indications (0-3%), the incidence was higher in nonmalignant recipients, ranging from 4-30%, and appears higher than the rates of graft failure rates reported in a large CIBTMR study of similar pediatric populations ( 129 – 142 , 144 ). The overall survival in pediatric hematologic malignant patients was 67-90%, 54% for adult, and 84-100% for non-malignant ( 129 – 142 ). The GRFS in pediatric patients was 50%-61% in hematologic malignancy patients and was 69-87% in the pediatric non-malignant cohort ( 129 – 142 ).…”

Section: Alpha/beta T Cell/b Cell Depletion (αßTb Depletion)mentioning

confidence: 62%

“…As expected with αß TB cell depletion, the rates of viral reactivation were higher than matched transplant approaches. Those reporting viral reactivations reported consistent rates of 40-65% of patients experiencing reactivations, with 20-65% reactivating CMV, 0-44% EBV, up to 57% adenovirus, 21% HHV6, and 23% BK virus ( 129 – 134 , 136 – 142 ). A large retrospective review showed the incidence of CMV and EBV reactivation were 53% and 33% respectively after αß TB cell depletion and associated with aGVHD ( 147 ).…”

Section: Alpha/beta T Cell/b Cell Depletion (αßTb Depletion)mentioning

confidence: 87%

“…The overall survival in pediatric hematologic malignant patients was 67-90%, 54% for adult, and 84-100% for non-malignant ( 129 – 142 ). The GRFS in pediatric patients was 50%-61% in hematologic malignancy patients and was 69-87% in the pediatric non-malignant cohort ( 129 – 142 ). In summary, this approach is best studied in pediatric HCT recipients with malignant and nonmalignant diseases, with good anti-leukemia effect, with particular attention paid to the risks of viral reactivations, which should include frequent viral infection surveillance and the plan for preventative or pre-emptive approaches.…”

Section: Alpha/beta T Cell/b Cell Depletion (αßTb Depletion)mentioning

confidence: 99%

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Controversies and expectations for the prevention of GVHD: A biological and clinical perspective

Watkins

Williams

2022

Front. Immunol.

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Severe acute and chronic graft versus host disease (GVHD) remains a major cause of morbidity and mortality after allogeneic hematopoietic cell transplantation. Historically, cord blood and matched sibling transplantation has been associated with the lowest rates of GVHD. Newer methods have modified the lymphocyte components to minimize alloimmunity, including: anti-thymocyte globulin, post-transplant cyclophosphamide, alpha/beta T cell depletion, and abatacept. These agents have shown promise in reducing severe GVHD, however, can be associated with increased risks of relapse, graft failure, infections, and delayed immune reconstitution. Nonetheless, these GVHD prophylaxis strategies have permitted expansion of donor sources, especially critical for those of non-Caucasian decent who previously lacked transplant options. This review will focus on the biologic mechanisms driving GVHD, the method by which each agent impacts these activated pathways, and the clinical consequences of these modern prophylaxis approaches. In addition, emerging novel targeted strategies will be described. These GVHD prophylaxis approaches have revolutionized our ability to increase access to transplant and have provided important insights into the biology of GVHD and immune reconstitution.

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Section: Alpha/beta T Cell/b Cell Depletion (αßTb Depletion)mentioning

confidence: 62%

Section: Alpha/beta T Cell/b Cell Depletion (αßTb Depletion)mentioning

confidence: 87%

Section: Alpha/beta T Cell/b Cell Depletion (αßTb Depletion)mentioning

confidence: 99%

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Controversies and expectations for the prevention of GVHD: A biological and clinical perspective

Watkins

Williams

2022

Front. Immunol.

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“…Several groups have since reported on successful use of TCRab+/CD19+ depleted transplant in various settings including for non-malignant diseases (76) including PIDs and Severe Combined Immune Deficiency (SCID) (77), Fanconi Anemia (78), and Sickle Cell Disease (79). Other trials have also confirmed efficacy in patients with malignancies (80,81).…”

Section: Tcrab+ T Cell-depletionmentioning

confidence: 99%

Selective depletion of naïve T cells by targeting CD45RA

Naik

2023

Front. Oncol.

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“…The patient received >8 × 10 6 /kg CD34 cells/kg, <1 × 10 5 /kg T cell receptor (TCR) αβ, and <2 × 10 5 /kg CD19 cells according to the pro to col in use at our cen ter. 31 No posttransplantation pharma co logic immu no sup pres sion was admin is tered.…”

Section: Clinical Case (Con Tin Ued)mentioning

confidence: 99%

Modern management of Fanconi anemia

Dufour

Pierri

2022

Hematology

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In this review, we present a clinical case report and discussion to outline the importance of long-term specific Fanconi anemia (FA) monitoring, and we discuss the main aspects of the general management of patients with FA and clinical complications. While several nontransplant treatments are currently under evaluation, hematopoietic stem cell transplantation (HSCT) remains the only therapeutic option for bone marrow failure (BMF). Although HSCT outcomes in patients with FA have remarkably improved over the past 20 years, in addition to the mortality intrinsic to the procedure, HSCT increases the risk and accelerates the appearance of late malignancies. HSCT offers the best outcome when performed in optimal conditions (moderate cytopenia shifting to severe, prior to transfusion dependence and before clonal evolution or myelodysplasia/acute myeloid leukemia); hence, an accurate surveillance program is vital. Haploidentical HSCT offers very good outcomes, although long-term effects on malignancies have not been fully explored. A monitoring plan is also important to identify cancers, particularly head and neck carcinomas, in very early phases. Gene therapy is still experimental and offers the most encouraging results when performed in early phases of BMF by infusing high numbers of corrected cells without genotoxic effects. Patients with FA need comprehensive monitoring and care plans, coordinated by centers with expertise in FA management, that start at diagnosis and continue throughout life. Such long-term follow-up is essential to detect complications related to the disease or treatment in this setting.

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Haploidentical Stem Cell Transplantation After TCR-αβ+ and CD19+ Cells Depletion In Children With Congenital Non-Malignant Disease

Cited by 13 publications

References 45 publications

Controversies and expectations for the prevention of GVHD: A biological and clinical perspective

Controversies and expectations for the prevention of GVHD: A biological and clinical perspective

Selective depletion of naïve T cells by targeting CD45RA

Modern management of Fanconi anemia

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