Official websites use. Share sensitive information only on official, secure websites. Box , Jerusalem , Israel. Email: azimran gmail. As of February 25, , patients had enrolled, the majority from Israel Median age at GOS entry was Most patients had type 1 GD A total of Hematological and visceral findings at the time of GOS entry were close to normal for most patients, probably a result of previous treatment; however, spleen volume of patients in Israel was almost double that of patients elsewhere 7.
This analysis aimed to provide an overview of GOS and present baseline demographic and disease characteristics of participating patients to help improve the understanding of the natural history of GD and inform the overall management of patients with the disease. GD may be regarded as a phenotypic continuum 2 owing to its highly variable clinical presentation; however, patients continue to be classified into the traditionally recognized clinical variants of GD, types 1, 2, or 3, to facilitate patient management.
This can help to better define the population distribution of the disease and its variability in presentation or severity, and assess response to treatment to inform management strategies. Here we provide an overview of GOS and present the baseline demographic and general disease characteristics of participating patients in the registry. This analysis also provides a baseline for additional analyses and makes comparisons between specific subpopulations within GOS. The management, administration, and infrastructure of GOS is funded by Shire.
The specific aims of GOS are to better characterize the global GD population, to monitor the natural history of treated and untreated patients with GD of any type, and to enhance understanding of the effectiveness and safety of current GD treatments through observing the evolving nature of the disease among patients treated to varying degrees and at different stages.
GOS is directed by a steering committee that includes international experts investigators and medical personnel from Shire. The steering committee meets on an annual basis to review the progress in the operation, database management, and analysis of GOS. Patients currently participating in ongoing clinical trials are excluded from enrollment. Patients can withdraw from the registry at any time for any reason without prejudice to their future care. The development of the eCRF involved 2 different vendors to optimize the final version for use in this registry.
