title: Phase I trial of low-dose interleukin 2 therapy in patients with Wiskott-Aldrich syndrome.
abstract: Low dose IL-2 can restore the function of T and NK cells from Wiskott-Aldrich (WAS) patients. However, the safety of in vivo IL-2 in WAS is unknown.
  
  
  
  A phase-I study to assess safety of low dose IL-2 in WAS.
  
  
  
  Patients received 5 daily subcutaneous IL-2 injections, every 2months, for three courses. A "3+3" dose escalation method was used.
  
  
  
  6 patients received the 0.5millionunits/m2/day dose without serious adverse events. However, 2 of 3 patients receiving the 1millionunits/m2/day dose developed thrombocytopenia requiring platelet transfusions. A statistically significant platelet increase occurred in patients receiving the 0.5millionunits/m2/day dose. A trend toward higher T, B and NK cell numbers and higher T regulatory cell percentages was observed.
  
  
  
  We have identified a safe IL-2 dose for WAS patients. Additional trials are indicated to study the efficacy of this immunostimulant as a therapy for WAS.
authors.affiliation: Division of Allergy and Immunology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States. Electronic address: Jyonouchi@email.chop.edu.
authors.forename: Soma
authors.initials: S
authors.lastname: Jyonouchi
authors.affiliation: The Children's Hospital of Philadelphia, Philadelphia, PA, United States.
authors.forename: Brenda
authors.initials: B
authors.lastname: Gwafila
authors.affiliation: Division of Immunology Allergy and Rheumatology, Departments of Pediatrics, Pathology and Immunology, Baylor College of Medicine, United States; Center for Human Immunobiology, Texas Children's Hospital, United States.
authors.forename: Lavesh A
authors.initials: LA
authors.lastname: Gwalani
authors.affiliation: Phoenix Children's Hospital, Phoenix, AZ, United States.
authors.forename: Maria
authors.initials: M
authors.lastname: Ahmad
authors.affiliation: The University of Minnesota Masonic Children's Hospital, Minneapolis, MN, United States.
authors.forename: Chistopher
authors.initials: C
authors.lastname: Moertel
authors.affiliation: Myers Clinic, Philippi, WV, United States.
authors.forename: Cecil
authors.initials: C
authors.lastname: Holbert
authors.affiliation: The Children's Hospital of Philadelphia, Philadelphia, PA, United States.
authors.forename: Ji Young
authors.initials: JY
authors.lastname: Kim
authors.affiliation: Roger Maris Cancer Center, Sanford Health, Fargo, ND, United States.
authors.forename: Nathan
authors.initials: N
authors.lastname: Kobrinsky
authors.affiliation: Division of Allergy and Immunology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States.
authors.forename: Sumita
authors.initials: S
authors.lastname: Roy-Ghanta
authors.affiliation: Division of Immunology Allergy and Rheumatology, Departments of Pediatrics, Pathology and Immunology, Baylor College of Medicine, United States; Center for Human Immunobiology, Texas Children's Hospital, United States.
authors.forename: Jordan S
authors.initials: JS
authors.lastname: Orange
mesh_terms: D000276:Adjuvants, Immunologic / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D000293:Adolescent; D001402:B-Lymphocytes / Q000276:immunology; D002648:Child; D002675:Child, Preschool; D006801:Humans; D007376:Interleukin-2 / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D007694:Killer Cells, Natural / Q000276:immunology; D007958:Leukocyte Count; D010976:Platelet Count; D013601:T-Lymphocytes / Q000276:immunology; D014923:Wiskott-Aldrich Syndrome / Q000097:blood / Q000188:drug therapy* / Q000276:immunology
chemical_list: D000276:Adjuvants, Immunologic; D007376:Interleukin-2
keywords: Low dose IL-2; Wiskott-Aldrich syndrome; X-linked thrombocytopenia