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Blood, 15 January 2002, Vol. 99, No. 2, pp. 698-701
BRIEF REPORT
Nonmyeloablative conditioning followed by transplantation of
genetically modified HLA-matched peripheral blood progenitor cells for
hematologic malignancies in patients with acquired
immunodeficiency syndrome
Elizabeth M. Kang,
Moniek de Witte,
Harry Malech,
Richard A. Morgan,
Sheila Phang,
Charles Carter,
Susan F. Leitman,
Richard Childs,
A. John Barrett,
Richard Little, and
John F. Tisdale
From the Molecular and Clinical Hematology Branch,
National Institute of Diabetes and Digestive and Kidney Diseases;
National Institute of Allergy and Infectious Diseases; National Human
Genome Research Institute; Department of Nursing and Department of
Transfusion Medicine, Clinical Center; National Heart, Lung, and Blood
Institute; and National Cancer Institute, National Institutes of
Health, Bethesda, MD.
To assess the safety and efficacy of nonmyeloablative
allogeneic transplantation in patients with HIV infection, a clinical protocol was initiated in patients with refractory hematologic malignancies and concomitant HIV infection. The results from the first
2 patients are reported. The indications for transplantation were
treatment-related acute myelogenous leukemia and primary refractory
Hodgkin disease in patients 1 and 2, respectively. Only patient 1 received genetically modified cells. Both patients tolerated the
procedure well with minimal toxicity, and complete remissions were
achieved in both patients, but patient 2 died of relapsed Hodgkin
disease 12 months after transplantation. Patient 1 continues in
complete remission with undetectable HIV levels and rising CD4 counts,
and with both the therapeutic and control gene transfer vectors
remaining detectable at low levels more than 2 years after
transplantation. These results suggest that nonmyeloablative allogeneic
transplantation in the context of highly active antiretroviral therapy
is feasible in patients with treatment-sensitive HIV infection.

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