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Bone marrow transplantation in Fanconi anemia using matched sibling donors.
TLDR
The addition of ATG to the transplant regimen of low-dose CY, TAI, and cyclosporin was responsible for improvement in the survival of FA patients undergoing BMT, and was associated with a low incidence of complications including GVHD.
Newborn Screening for Severe Combined Immunodeficiency; The Wisconsin Experience (2008–2011)
TLDR
The T-cell receptor excision circle assay is a sensitive and specific test to identify infants with severe combined immunodeficiency and severe T- cell lymphopenia that leads to life-saving therapies such as hematopoietic stem cell transplantation prior to the acquisition of severe infections.
Bone marrow transplantation for T−B− severe combined immunodeficiency disease in Athabascan-speaking native Americans
TLDR
The results demonstrate the efficacy of BMT in treating infants with this distinct form of SCID, although B cell reconstitution remains a problem even with HLA-matched donors.
Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study.
TLDR
Although newborn screening has been effective in diagnosing SCID patients early in life, there is an urgent need to identify validated approaches through prospective trials to ensure that patients proceed to HCT infection free.
Autoimmune hemolytic anemia in patients with SCID after T cell-depleted BM and PBSC transplantation
TLDR
Delayed reconstitution of T cell immunity, due to T cell depletion, immunosuppressive conditioning and CsA, as well as paucity of regulatory T cells, are the likely explanations for the occurrence of autoimmune hemolytic anemia in patients with SCID who underwent a T cell-depleted haploidentical transplant.
Cognitive and adaptive behavior 1 and 3 years following bone marrow transplantation
TLDR
IQ at the 1 year follow-up was significantly lower than baseline and scores on the Vineland Adaptive Behavior Scales dropped significantly, but did not change between the1 year and 3 year evaluations.
Unrelated donor bone marrow transplantation for children with acute myeloid leukemia beyond first remission or refractory to chemotherapy.
TLDR
Children who underwent transplantation in remission had a superior outcome compared with children who underwent transplants during relapse or persistent disease, suggesting that unrelated donor bone marrow transplantation is an effective therapy in a significant proportion of children with recurrent or primary refractory AML.
SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery.
TLDR
A retrospective analysis of patients with severe combined immunodeficiency who received a hematopoietic cell transplantation as first-line treatment between 1982 and 2012 in 33 North American institutions found that Infection-free status and younger age at HCT were associated with improved survival.
Antitumor activity of hu14.18-IL2 in patients with relapsed/refractory neuroblastoma: a Children's Oncology Group (COG) phase II study.
TLDR
Patients with disease evaluable only by MIBG and/or BM histology had a 21.7% CR rate to hu14.8-IL2, whereas patients with bulky disease did not respond and warrants further testing in children with nonbulky high-risk neuroblastoma.
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