Problems to be Addressed
Dupuytren disease is the most common inherited disorder to affect connective tissues. Dupuytren disease is a chronic progressive disease which primarily affects the hands. The end result for many is hand deformities. These are treated by surgeons, but results are temporary, and repeat treatments progressively
Dupuytren disease is a chronic progressive disease which primarily affects the hands. The end result for many is hand deformities. These are treated by surgeons, but results are temporary, and repeat treatments progressively more risky and less effective. There is currently no medical cure.
Surgery has reached the theoretic limit of disease control: recurrence after dermofasciectomy occurs at the same rate as progression to contracture in untreated patients with the disease but no contracture. There has been no progress in long-term control in the last fifty years.
Move the focus from procedure based treatment to biologic disease-modifying treatment of the root cause of the disease.
Progress in the development of a medical cure of Dupuytren disease may also provide new treatments for Ledderhose, Peyronie, and frozen shoulder, and other fibrotic conditions.
Historical Roadblocks to Dupuytren Research
- It’s common. The Dupuytren genetic risk pool includes at least one-quarter of Caucasians, reducing the likelihood of identifying a unique molecular target with analysis of unaffected tissues.
- It’s slow. The risk of progression from nodule to contracture requiring treatment is less than 2 percent per year. The effectiveness of prophylactic treatment requires large, long duration studies to have adequate statistical power.
- It’s complicated. Gene expression studies have identified a complex set of abnormalities which have not yet been simplified enough to identify the best molecular target.
- It’s unpredictable Clinical studies are hampered by the lack of adequate data on natural history and by great individual variability without a reliable marker of biological aggressiveness. Until this is resolved, clinical studies must be on a larger scale than have been previously reported.
- It’s spread out. No single center currently treats a large number of Dupuytren patients with the full range of treatment options. Data must be pooled from multiple sources.
- Registry studies are difficult. Paperwork, time, cost and aggravation of multicenter clinical registry studies make them burdensome and difficult to maintain.
|Genetic and longitudinal clinical study||Patients will be engaged through direct-to-patient enrollment. Online surveys will be used to collect longitudinal clinical data. A small fraction of the 10 million Americans with Dupuytren disease will populate a large study, which will be expanded globally as needed. The existing technology of secure, web-based, HIPAA compliant patient surveys in use by the National Databank for Rheumatic Diseases will provide the backbone for clinical data collection. A central IRB will provide oversight for blood collection to a central biobank. Use of existing web-based data collection methods and minimizing the administrative burden to participating surgeons will result in cost reduction compared to traditional multicenter studies. Analysis of large-scale clinical and genomic data will define unique biomarkers and RNA or proteomic targets for therapeutic drug development.|
|Large sample size|
|Use existing tools and technology|
|Least disruption of normal practice|
|Cost efficient design|
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The Dupuytren Foundation is open to inquiries by potential collaborators for this project.