Marfan's Syndrome

Diagnosis and Treatment Center in Haiti’s Central Plateau

At first anecdotally, and over the past three years, with careful documentation of clinical criteria, medical students from the University of Miami and Tauro Osteopathic Medical School have documented more than 100 patients, mostly children, with Marfan's Syndrome in isolated communities in Haiti's Central Plateau. The prevalence of Marfan's Syndrome appears to range from 5% to an astounding 25% of the children who attend mobile clinics in these communities.

This initial identification of index cases only scratches the surface of the problem, as the US teams have only visited eight of the 30 sites were mobile clinics are conducted in have not been able to assess parents and siblings born subsequent to their visits (attendance of children in mobile clinics is mandatory while attendance of adults is not). Treatment with antihypertensive medicines has now been shown to delay or prevent dilatation of the aorta, and the dreaded complications of Marfan's Syndrome – aortic insufficiency or dissecting aneurysm. The advent of effective treatment, therefore, creates a need – indeed, a moral imperative – to identify and treat each patient in Haiti’s central plateau afflicted with Marfan's Syndrome.

The Opportunity

The infrastructure and workforce provided by Medishare for Haiti, the clinical experiences and expertise of the Miller school and Tauro faculty, coupled with an already identified cohort of over 100 patients with Marfan's syndrome creates an ideal opportunity to establish an international Marfan's Syndrome Diagnosis and Treatment Center in Haiti's Central Plateau. In addition to the impact of this program in establishing a US standard of care for patients with Marfan's syndrome in the poorest country in the Western Hemisphere, The Center would also provide a platform for research centered on diagnosis, treatment and genetics of the syndrome.

It should be noted that in addition to the resources described in the background section, the University of Miami and its affiliates also have a guest house and conference center to host visiting Foundation members, clinicians and researchers. There are three donated echocardiogram machines , 4 ambulances and 20 motorcycles to facilitate patient transportation. These resources will be donated as in-kind contributions to the project. We also have pharmacies and pharmacists which can supply medications and calculate the doses of medicines for children in terms of milligrams per kilogram. Finally, the University of Miami will have the capacity to perform elective cardiac surgery by September2015– both at its affiliated hospital in Port-au-Prince – Centre Bernard Mevs , which has operating rooms, neonatal and surgical intensive care units and pediatric residents and faculty providing 24/7 coverage, as well as the potential to fly patients to Miami to be cared for at Miami's teaching hospitals. The University of Miami is currently crafting a business plan to establish and sustain a brand-new trauma hospital to be located in Port-au-Prince within two years.

The Proposal

It is proposed that the Marfan's Foundation, The University Miami Miller School of Medicine and its Nongovernmental Organization affiliates enter into a cooperative agreement to establish a Marfan's Syndrome diagnosis, treatment and research program in Haiti central plateau. The program would be established in three phases:

Phase 1

To begin immediately upon execution of a Memorandum of Agreement – collaborative planning, networking, fundraising and training of the Haitian workforce in diagnosis, assessment (including sonography) and treatment of Marfan's syndrome. Already established contacts with the NBA, the National Retired Basketball Players Associationand pharmaceutical companies should facilitate fundraising.

Phase 2

Tto begin when sufficient funds are raised to assure the successful implementation and operation of the program for a minimum of three years (estimated at approximately$250,000 – $300,000 per year). During phase 2 we will establish a database of currently identified cases, including aortic root dimensions and initiate treatment according to currently accepted best practices. With the workforce trained during phase 1, screening using clinical criteria will be extended to all 30 "mobile clinic" communities and genograms established for all index cases and begin primary-care and treatment as appropriate.