NAME: Study of Gamma Interferon in Chronic Granulomatous Disease (CGD.DAT) SIZE: 203 observations, 16 variables. SOURCE: Counting Processes and Survival Analysis by T. Fleming & D. Harrington (1991), John Wiley & Sons Publishers DESCRIPTIVE ABSTRACT: The following data are from a placebo controlled randomized trial of gamma interferon in chronic granulotomous disease (CGD). The CGD study, which is described in a report by the International CGD Cooperative Study Group (1991), was designed to have a single interim analysis when the follow- up data as of July 15, 1989 were complete. The monitoring committee for the trial terminated the trial at a meeting on September 22, 1989, based on the interim analysis. The treatment given each patient was unblinded at the first scheduled visit for that patient following the decision of the monitoring committee. The data for each case give the time to initial and any recurrent serious infections, from study entry until the first scheduled visit of the patient after the decision of the monitoring committee. These infections are those observed through the interim analysis date of record (7/15/89) as well as the additional data on occurrence of serious infections between the interim analysis cutoff and the final blinded study visit for each patient. There is a minimum of 1 record per patient, with an additional record for each serious infection occurring up to the study completion date. LIST OF VARIABLES: Variable Description _________________________________________________________________________ ID Case identification number. The first 3 digits denote a hospital code. RDT Date randomized onto study (for S = 1 record), in the format mmddyy. IDT Date of onset of a serious infection, or date the patient was taken off the study. Zl Treatment Code, 1 = Gamma Interferon, 2 = placebo. Z2 Pattern of inheritance, 1 = X-linked, 2 = autosomal recessive. Z3 Age, in years. Z4 Height, in cm. Z5 Weight, in kg. Z6 Using corticosteroids at time of study entry, 1 = yes, 2 = no. Z7 Using prophylactic antibiotics at time of study entry, 1 = yes, 2 = no. Z8 Gender, 1 = male, 2 = female. Z9 Hospital category, 1 = US - NIH, 2 = US - other, 3 = Europe-Amsterdam, 4 = Europe-other. Tl Elapsed time (in days) from randomization (from S = 1 record) to diagnosis of a serious infection, or if a censored observation, elapsed time from randomization to censoring date. Computed as IDT-RDT (from S = 1 record). T2 0, for S = 1. If S > 1, T2 = Tl (from previous record) + 1. D Censoring indicator, 1 = Non-censored observation (i.e., a serious infection occurred at the date specified by the IDT field), 2 = censored observation (i.e., the patient was taken off study at the date specified by the IDT field.) Note that only patient ID = 238087 had a serious infection diagnosed on the date he was taken off study. S Sequence number. For each patient, the infection records are in sequence number order. ________________________________________________________________________________ STORY BEHIND THE DATA: Chronic Granulomatous Disease (CGD) is a group of inherited rare disorders of the immune function characterized by recurrent pyogenic infections which usually present early in life and may lead to death in childhood. Phagocytes from CGD patients ingest microorganisms normally but fail to kill them, primarily due to the inability to generate a respiratory burst dependent on the production of superoxide and other toxic oxygen metabolites. Thus, it is the failure to generate microbicidal oxygen metabolites within the phagocytes of CGD patients which confers the greatly increased susceptibility to these severe or even life threatening infections. There is evidence establishing a role for gamma interferon as an important macrophage activating factor which could restore superoxide anion production and bacterial killing by phagocytes in CGD patients. In order to study the ability of gamma interferon to reduce the rate of serious infections, that is, the rate of infections requiring hospitalization for parenteral antibiotics, a double-blinded clinical trial was conducted in which patients were randomized to placebo vs. gamma interferon. Between October 1988 and March 1989, 128 eligible patients with CGD were accrued by the International CGD Cooperative Study Group. Since the study required delivering placebo injections three times weekly for a twelve month period to one-half of the patients, most being children, there was particular interest in achieving early termination of the trial if early results were extreme. A single interim analysis was to be performed as soon as patient follow-up was available through July 1989, six-months after the date on which one-half of the patients had been accrued. At the time of interim analysis, twenty of 65 placebo patients and seven of 63 patients on gamma interferon each had experienced at least one serious infection