Radiosensitive severe combined immunodeficiency disease

CC Dvorak, MJ Cowan - Immunology and Allergy …, 2010 - immunology.theclinics.com
Patients with severe combined immunodeficiency (SCID) disease have classically been
divided into those with residual B cells (TÀB+ phenotype) and those whose defects produce …

Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID)

B Chan, D Wara, J Bastian, MS Hershfield… - Clinical …, 2005 - Elsevier
Adenosine deaminase (ADA)-deficient Severe Combined Immunodeficiency (ADA-deficient
SCID) is characterized by impaired lymphocyte development and function resulting from the …

Nonmyeloablative stem cell transplantation for congenital immunodeficiencies

P Amrolia, HB Gaspar, A Hassan… - Blood, The Journal …, 2000 - ashpublications.org
The optimal approach for stem cell transplantation in children with immunodeficiency has yet
to be determined. Conditioning therapy is necessary for reliable engraftment and full …

Long-term T-cell reconstitution after hematopoietic stem-cell transplantation in primary T-cell–immunodeficient patients is associated with myeloid chimerism and …

M Cavazzana-Calvo, F Carlier, F Le Deist, E Morillon… - Blood, 2007 - ashpublications.org
We studied T-cell reconstitution in 31 primary T-cell–immunodeficient patients who had
undergone hematopoietic stem-cell transplantation (HSCT) over 10 years previously. In 19 …

Bone marrow transplantation for T− B− severe combined immunodeficiency disease in Athabascan-speaking native Americans

AS O'Marcaigh, K DeSantes, D Hu, H Pabst… - Bone marrow …, 2001 - nature.com
A distinct form of autosomal recessive T− B− severe combined immunodeficiency disease
occurs with a high frequency among Athabascan-speaking Native Americans (SCIDA) …

A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency

CC Dvorak, BN Horn, JM Puck… - Pediatric …, 2014 - Wiley Online Library
For infants with SCID, the ideal conditioning regimen before allogeneic HCT would omit
cytotoxic chemotherapy to minimize short‐and long‐term complications. We performed a …

Outcome of boost haemopoietic stem cell transplant for decreased donor chimerism or graft dysfunction in primary immunodeficiency

MA Slatter, A Bhattacharya, M Abinun… - Bone Marrow …, 2005 - nature.com
Haemopoietic stem cell transplants (HSCT) cure increasing numbers of primary
immunodeficiencies (PID): residual recipient T-cell function increases risk of incomplete or …

Long-term outcome of non-ablative booster BMT in patients with SCID

CL Teigland, RE Parrott, RH Buckley - Bone marrow transplantation, 2013 - nature.com
SCID is a fatal syndrome caused by mutations in at least 13 different genes. It is
characterized by the absence of T cells. Immune reconstitution can be achieved through …

CAMPATH-1M T-cell depleted BMT for SCID: long-term follow-up of 19 children treated 1987-98 in a single center

AR Gennery, AM Dickinson, K Brigham, D Barge… - …, 2001 - Taylor & Francis
Background: SCID can be cured by BMT. Depletion of mature T cells from BM has enabled
HLA non-identical stem-cell transplantation. We report the outcome of 30 patients treated …

Bone marrow transplantation in severe combined immunodeficiency from a sibling who had received a paternal bone marrow transplant

ER Stiehm, RL Roberts, J Hanley-Lopez… - … England Journal of …, 1996 - Mass Medical Soc
Bone marrow transplantation to treat autosomal recessive severe combined
immunodeficiency was undertaken in a one-month-old girl. The donor was the patient's HLA …