|
|
 Previous Article | Table of Contents | Next Article 
Blood, 1 February 2002, Vol. 99, No. 3, pp. 872-878
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Hematopoietic stem cell transplantation for severe combined
immunodeficiency in the neonatal period leads to superior thymic output
and improved survival
Laurie A. Myers,
Dhavalkumar D. Patel,
Jennifer
M. Puck, and
Rebecca H. Buckley
From Duke University Medical Center, Durham, NC, and
the National Human Genome Research Institute, National Institutes of
Health, Bethesda, MD.
All genetic types of severe combined immunodeficiency
(SCID) can be cured by stem cell transplantation from related donors. The survival rate approaches 80%, and most deaths result from opportunistic infections acquired before transplantation. It was hypothesized that the survival rate and kinetics of immune
reconstitution would be improved for infants receiving
transplants in the neonatal period (first 28 days of life),
prior to the development of infections. A 19.2-year
retrospective/prospective analysis compared immune function in 21 SCID
infants receiving transplants in the neonatal period with that in 70 SCID infants receiving transplants later. Lymphocyte phenotypes,
proliferative responses to mitogens, immunoglobulin levels, and
T-cell antigen receptor excision circles (TRECs) were measured before
transplantation and sequentially after transplantation. Of 21 SCID
infants with transplantations in the neonatal period, 20 (95%)
survive. Neonates were lymphopenic at birth (1118 ± 128 lymphocytes
per cubic millimeter). Infants receiving transplants early developed
higher lymphocyte responses to phytohemagglutinin and higher numbers of
CD3+ and CD45RA+ T cells in the first 3 years of life than those receiving transplants late
(P < .05). TRECs peaked earlier and with higher values
(P < .01) in the neonatal transplantations (181 days to
1 year) than in the late transplantations (1 to 3 years). SCID
recipients of allogeneic, related hematopoietic stem cells in the
neonatal period had higher levels of T-cell reconstitution and thymic
output and a higher survival rate than those receiving transplants
after 28 days of life. An improved outcome for this otherwise fatal syndrome could be achieved with newborn screening for lymphopenia so
that transplantation could be performed under favorable thymopoietic conditions.
 CiteULike  Connotea  Del.icio.us  Digg  Reddit  Technorati What's this?
This article has been cited by other articles:
|
|
|
|
 
M. Sarzotti-Kelsoe, C. M. Win, R. E. Parrott, M. Cooney, B. K. Moser, J. L. Roberts, G. D. Sempowski, and R. H. Buckley
Thymic output, T-cell diversity, and T-cell function in long-term human SCID chimeras
Blood,
August 13, 2009;
114(7):
1445 - 1453.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
K. Liuba, C. J. H. Pronk, S. R. W. Stott, and S.-E. W. Jacobsen
Polyclonal T-cell reconstitution of X-SCID recipients after in utero transplantation of lymphoid-primed multipotent progenitors
Blood,
May 7, 2009;
113(19):
4790 - 4798.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
A. Aiuti, F. Cattaneo, S. Galimberti, U. Benninghoff, B. Cassani, L. Callegaro, S. Scaramuzza, G. Andolfi, M. Mirolo, I. Brigida, et al.
Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
N. Engl. J. Med.,
January 29, 2009;
360(5):
447 - 458.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
J. Chinen, J. Davis, S. S. De Ravin, B. N. Hay, A. P. Hsu, G. F. Linton, N. Naumann, E. Y. H. Nomicos, C. Silvin, J. Ulrick, et al.
Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency
Blood,
July 1, 2007;
110(1):
67 - 73.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
M. Cavazzana-Calvo, F. Carlier, F. Le Deist, E. Morillon, P. Taupin, D. Gautier, I. Radford-Weiss, S. Caillat-Zucman, B. Neven, S. Blanche, et al.
Long-term T-cell reconstitution after hematopoietic stem-cell transplantation in primary T-cell-immunodeficient patients is associated with myeloid chimerism and possibly the primary disease phenotype
Blood,
May 15, 2007;
109(10):
4575 - 4581.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
N. S. Green and T. H. Murray
GREEN AND MURRAY RESPOND
Am J Public Health,
April 1, 2007;
97(4):
589 - 590.
[Full Text]
[PDF]
|
|
|
|
|
|
|
J. A. Borghans, R. G. Bredius, M. D. Hazenberg, H. Roelofs, E. C. Jol-van der Zijde, J. Heidt, S. A. Otto, T. W. Kuijpers, W. E. Fibbe, J. M. Vossen, et al.
Early determinants of long-term T-cell reconstitution after hematopoietic stem cell transplantation for severe combined immunodeficiency
Blood,
July 15, 2006;
108(2):
763 - 769.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
A. J. Thrasher, S. Hacein-Bey-Abina, H. B. Gaspar, S. Blanche, E. G. Davies, K. Parsley, K. Gilmour, D. King, S. Howe, J. Sinclair, et al.
Failure of SCID-X1 gene therapy in older patients
Blood,
June 1, 2005;
105(11):
4255 - 4257.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
M. Schmidt, S. Hacein-Bey-Abina, M. Wissler, F. Carlier, A. Lim, C. Prinz, H. Glimm, I. Andre-Schmutz, C. Hue, A. Garrigue, et al.
Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial
Blood,
April 1, 2005;
105(7):
2699 - 2706.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
K. Rao, P. J. Amrolia, A. Jones, C. M. Cale, P. Naik, D. King, G. E. Davies, H. B. Gaspar, and P. A. Veys
Improved survival after unrelated donor bone marrow transplantation in children with primary immunodeficiency using a reduced-intensity conditioning regimen
Blood,
January 15, 2005;
105(2):
879 - 885.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
M. Sarzotti, D. D. Patel, X. Li, D. A. Ozaki, S. Cao, S. Langdon, R. E. Parrott, K. Coyne, and R. H. Buckley
T Cell Repertoire Development in Humans with SCID After Nonablative Allogeneic Marrow Transplantation
J. Immunol.,
March 1, 2003;
170(5):
2711 - 2718.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
L R Wedderburn, M Abinun, P Palmer, and H E Foster
Autologous haematopoietic stem cell transplantation in juvenile idiopathic arthritis
Arch. Dis. Child.,
March 1, 2003;
88(3):
201 - 205.
[Full Text]
[PDF]
|
|
|
|
|
|
|
M. D. Cooper, L. L. Lanier, M. E. Conley, and J. M. Puck
Immunodeficiency Disorders
Hematology,
January 1, 2003;
2003(1):
314 - 330.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
