Ulrich Pannicke.

Patient 1 became ill at 1 month of age with oral candidiasis and feeding difficulties. At 4 months of age, she had Escherichia coli septicemia, parainfluenza virus type 1 pneumonia, and persistent oral candidiasis. Laboratory assessment showed hypogammaglobulinemia , and SCID was diagnosed clinically. Her condition was stabilized with antimicrobial agents and intravenous immune globulin. She received a cord-blood transplant after reduced-intensity conditioning, however the graft was rejected. She experienced two episodes of pneumococcal bacteremia connected with osteomyelitis, from which she recovered, but she died from Mycobacterium avium sepsis. There is no genealogy of immunodeficiency or loss of life in early infancy.This difference was not anticipated and could have already been due to chance. We obtained primary-outcome information for more than 99 percent of sufferers and validated vital status. However, we didn’t perform follow-up examinations, and our telephone ascertainment of functional outcomes was at the mercy of possible miscommunication, poorly informed proxy respondents, and recording mistakes. Although we did not validate patients’ ability to walk, in cases where both individuals and their proxies answered the relevant question about walking ability, we found strong agreement between your two reports.