Hematopoietic stem cell transplantation for primary immunodeficiency disease

Dvorak, Christopher C.; Cowan, Morton J.

doi:10.1038/sj.bmt.1705890

Cited by 66 publications

(54 citation statements)

References 37 publications

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“…For nonprimary immunodeficiency patients, at the 12-week evaluation, if the absolute CD4 count was less than 100 cells/mm 3 and there was no evidence of active GVHD, a prophylactic infusion of 3 Â 10 4 cryopreserved donor CD3 þ cells/kg was performed. For primary immunodeficiency patients, DLI could be administered as early as 4 weeks post-HSCT if there were no detectible CD4 þ cells.…”

Section: Donor Pbsc Collection and Manipulationmentioning

confidence: 99%

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Clinical and immunologic outcomes following haplocompatible donor lymphocyte infusions

Dvorak

Gilman

Horn

et al. 2009

Bone Marrow Transplant

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We retrospectively analyzed the characteristics of 16 consecutive pediatric patients who received one or more G-CSF-mobilized donor lymphocyte infusions (DLI) following a T-cell-depleted haplocompatible hematopoietic SCT (HSCT) to enhance immune recovery and/ or treat an infection. The median time from HSCT to administration of first DLI was 12 weeks and the median dose of DLI administered was 3 Â 10 4 /kg (range, 2.5-6 Â 10 4 /kg). The incidence of Grade I-II acute GVHD was 19% (95% confidence interval (CI), 6-44%), and there were no cases of Grade III-IV acute GVHD. Chronic GVHD developed in 13% (95% CI, 2-37%) of patients. In surviving patients who did not undergo a second stem cell infusion, T-cell numbers and function increased to a protective level in a median of 3 months (range, 2-12.5 months) following the first DLI administration. In patients given DLI for treatment of an infection, 75% (95% CI, 46-92%) cleared their infection after a median of 9 weeks (range, 1-27 weeks). In patients with CMV infection, the development of CMV-specific T cells was observed following DLI. The 1-year overall survival following haplocompatible DLI was 71% (95% CI, 59-83%), with a median follow-up of 16 months from the first DLI.

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Section: Donor Pbsc Collection and Manipulationmentioning

confidence: 99%

“…3 Haplocompatible donors share a single HLA haplotype with the transplant recipient. Due to the HLA disparities involved, most haplocompatible HSCT protocols utilize ex vivo T-cell depletion to avoid severe GVHD.…”

Section: Introductionmentioning

confidence: 99%

Clinical and immunologic outcomes following haplocompatible donor lymphocyte infusions

Dvorak

Gilman

Horn

et al. 2009

Bone Marrow Transplant

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“…Selection of a suitable donor by HLA matching and/or an appropriate conditioning regimen has improved the outcome of HCT for leukemia patients 1 and PID patients. 2,3 Recently, successful outcomes of cord blood transplantation (CBT) and BM transplantation (BMT) have been observed even in HLAmismatched conditions. [4][5][6][7][8] Despite these improved outcomes, transplantation-related morbidities such as graft failure, GVHD and infection are still major problems that affect the prognosis and/or quality of life.…”

Section: Introductionmentioning

confidence: 99%

Cord blood transplantation is associated with rapid B-cell neogenesis compared with BM transplantation

Nakatani

Imai

Shigeno

et al. 2014

Bone Marrow Transplant

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Hematopoietic cell transplantation (HCT) is used for treatment of hematopoietic diseases. Assessment of T-and B-cell reconstitution after HCT is crucial because poor immune recovery has a major effect on the clinical course. In this study, we retrospectively analyzed T-cell receptor excision circles (TRECs) as well as signal and coding joint kappa-deleting recombination excision circles (sjKRECs and cjKRECs, respectively) as markers of newly produced lymphocytes in 133 patients (56 primary immunodeficient and 77 malignant cases, median (range): 12 (0-62) years old). We analyzed the kinetics of TREC and KREC recovery and determined the factors that contributed to better immune recovery. KRECs became positive earlier than TRECs and increased thereafter. Younger recipient age had a favorable effect on recovery of sjKRECs and cjKRECs. Compared with BM and peripheral blood, our data suggested that cord blood (CB) provided rapid B-cell recovery. CB also provided better B-cell neogenesis in adult HCT recipients. Chronic GVHD was associated with low TRECs, but not increased sjKRECs/cjKRECs. Finally, positive sjKRECs 1 month after HCT were associated with fewer infectious episodes. Monitoring of TRECs and KRECs may serve as a useful tool for assessment of immune reconstitution post HCT.

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“…2,4 The optimal approach to hematopoietic SCT in children with PID who lack a matched sibling donor is controversial. 5 Some centers continue to use myeloablative conditioning regimen in children with PID who undergo HLA-mismatched related T-cell-depleted BMT associated with high risk of graft failure. 2,5 However, recent success with reduced-intensity conditioning regimen before unrelated BMT shows that this may provide better engraftment, immune reconstitution and survival for patients with PID than the previous alternative.…”

mentioning

confidence: 99%

“…5 Some centers continue to use myeloablative conditioning regimen in children with PID who undergo HLA-mismatched related T-cell-depleted BMT associated with high risk of graft failure. 2,5 However, recent success with reduced-intensity conditioning regimen before unrelated BMT shows that this may provide better engraftment, immune reconstitution and survival for patients with PID than the previous alternative. 6,7 Very few patients with this pediatric emergency have undergone transplantation choosing umbilical cord blood as a first choice of hematopoietic stem cell source, despite the significantly faster availability of banked cryopreserved umbilical cord blood units and the higher frequency of rare haplotypes compared with BM registries.…”

mentioning

confidence: 99%

Unrelated cord blood transplantation and reduced-intensity conditioning regimen for graft failure in a child with Major Histocompatibility Complex class II deficiency

Bonduel

Staciuk

Figueroa

et al. 2008

Bone Marrow Transplant

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Hematopoietic stem cell transplantation for primary immunodeficiency disease

Cited by 66 publications

References 37 publications

Clinical and immunologic outcomes following haplocompatible donor lymphocyte infusions

Clinical and immunologic outcomes following haplocompatible donor lymphocyte infusions

Cord blood transplantation is associated with rapid B-cell neogenesis compared with BM transplantation

Unrelated cord blood transplantation and reduced-intensity conditioning regimen for graft failure in a child with Major Histocompatibility Complex class II deficiency

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