Recombinant interleukin 2 therapy in severe combined immunodeficiency disease

R. Pahwa, T. Chatila, S. Pahwa, C. Paradise, N. K. Day, R. Geha, S. A. Schwartz, H. Slade, N. Oyaizu, R. A. Good

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Abstract

Severe combined immunodeficiency disease (SCID) is a congenital disorder of severe B- and T-lymphocyte dysfunction in which several pathogenic mechanisms have been identified. The present study describes a female child with SCID who had a primary defect in the ability of T cells to secrete interleukin 2 (IL-2). B- and T-cell numbers were normal, but their functions were severely deficient. Mitogen and antigen-driven lymphoproliferative responses were diminished but were correctable in vitro with recombinant IL-2 (rIL-2). The patient's phytohemagglutinin-stimulated lymphocytes expressed IL-2 receptors normally. Despite the presence of the gene for IL-2, the patient's cells were grossly deficient in messenger RNA for IL-2 and endogenous IL-2 production. Pokeweed mitogen-driven B-cell differentiation was decreased and was not corrected by the addition of normal T cells to the B cells. Two attempts at immune reconstitution by haploidentical bone marrow transplantation failed. Therapy with rIL-2 (30,000 units/kg, given daily i.v.) resulted in marked clinical improvements as well as improved T-cell functions. The child, now 3 yr old, has been on rIL-2 therapy for 2 yr and receives rIL-2 (30,000 units/kg) three times weekly at home. This case study points to a new direction in the treatment of such disorders with rIL-2.

Original languageEnglish (US)
Pages (from-to)5069-5073
Number of pages5
JournalProceedings of the National Academy of Sciences of the United States of America
Volume86
Issue number13
DOIs
StatePublished - 1989

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    Pahwa, R., Chatila, T., Pahwa, S., Paradise, C., Day, N. K., Geha, R., Schwartz, S. A., Slade, H., Oyaizu, N., & Good, R. A. (1989). Recombinant interleukin 2 therapy in severe combined immunodeficiency disease. Proceedings of the National Academy of Sciences of the United States of America, 86(13), 5069-5073. https://doi.org/10.1073/pnas.86.13.5069