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

Research output: Contribution to journalArticlepeer-review

81 Scopus citations


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
Issue number13
StatePublished - 1989
Externally publishedYes

ASJC Scopus subject areas

  • General


Dive into the research topics of 'Recombinant interleukin 2 therapy in severe combined immunodeficiency disease'. Together they form a unique fingerprint.

Cite this