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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. Expand
An intermediate alemtuzumab schedule reduces the incidence of mixed chimerism following reduced-intensity conditioning hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis.
TLDR
The findings indicate that intermediate RIC reduces the incidence of mixed chimerism, is associated with a low incidence of upfront acute GVHD, and decreases the need for additional hematopoietic cell products after HCT. Expand
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. Expand
Long‐term outcomes of 176 patients with X‐linked hyper‐IgM syndrome treated with or without hematopoietic cell transplantation
TLDR
No difference in survival was observed between patients treated with or without HCT across all diagnosis years (1964‐2013), however, survivors treated with HCT experienced somewhat greater well‐being, and hazards associated with H CT decreased, reaching levels of significantly less risk in the late 1990s. Expand
Allogeneic hematopoietic stem cell transplantation for severe, refractory juvenile idiopathic arthritis.
TLDR
All patients with juvenile idiopathic arthritis had significant improvement of arthritis, resolution of MAS, and improved quality of life early following allo-HSCT; most importantly, 11 children achieved complete drug-free remission at the last follow-up. Expand
Experience with Alemtuzumab, Fludarabine, and Melphalan Reduced-Intensity Conditioning Hematopoietic Cell Transplantation in Patients with Nonmalignant Diseases Reveals Good Outcomes and That the
TLDR
A retrospective study of 206 patients with metabolic diseases, non-Fanconi anemia marrow failure disorders, and primary immune deficiencies who underwent 210 consecutive RIC HCT procedures at Cincinnati Children's Hospital concludes that alemtuzumab, fludarabine, and melphalan Ric HCT offers good results for many patients and that the risk of developing mixed chimerism is influenced by underlying diagnosis, graft source, and alem TZM dosing. Expand
Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype
TLDR
A Reduced Intensity Conditioning (RIC) Phase I/II Pilot Study on Gene Transfer in Patients with SCA with a modified γ-Globin LV will be safe, feasible and efficacious, and be implementable in many transplant centers, including those in some of the resource-poor countries. Expand
Pretransplant Absolute Lymphocyte Counts Impact the Pharmacokinetics of Alemtuzumab.
TLDR
Almost all patients have persistence of lytic levels of alemtuzumab beyond day 0, at levels in excess of that needed to reduce the risk of acute graft-versus-host disease, and levels correlate with pretransplant ALC. Expand
K-Cl Cotransporter Gene Expression during Human and Murine Erythroid Differentiation*
TLDR
The results suggest that KCC3 is the dominant isoform in erythrocytes, with variable expression of KCC1 and KCC4 among individuals that could result in modulation of K CC activity. Expand
Prevalence of oral and systemic manifestations in pediatric HIV cohorts with and without drug therapy.
TLDR
The prevalence of oral and systemic manifestations is a persistent feature associated with pediatric HIV, though of moderate intensity in those using HAART and may vary according to individual immune status. Expand
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