Dr. Sylvia Titi Singer and co-investigators (Drs. Elliott Vichinsky, Frans Kuypers, and Howard Rosenfeld at Children’s Hospital & Research Center at Oakland; Drs. Jeff Fineman and Teresa De Marco at University of California San Francisco; and Dr. Diane Nugent at Children’s Hospital Orange County) have an open study aimed at assessing causes for the development of pulmonary hypertension (PHT) in thalassemia and based on these findings, intervening with specific treatment.
This is a five-year study which looks at risk factors for developing PHT and assesses the effects of two different treatments on the identified risk factors. PHT is increasingly recognized as a cause for significant morbidity and mortality in patients with thalassemia and is still frequently under-diagnosed because most patients do not undergo routine monitoring of right heart pressure, which detects PHT. In addition, the risk factors and causes are not fully understood. Therfore the best treatment approach is not well defined.
This study aims to both determine that increased coagulation and platelet activation are prime causes for the development of PHT in thalassemia and to institute a pilot intervention trial in patients with PHT.
From this study, the patient will receive information about the the presence of clinical or laboratory markers that put him or her at higher risk for developing PHT or the presence of PHT as seen on echocardiogram.
Who can participate?
- Chronically transfused or non-transfused patients with alpha-thalassemia or beta- thalassemia
- Patients over the age of 10 years
- Patients taking aspirin or Coumadin for at least 10 days prior to the laboratory assessment
- Recipients of successful bone marrow transplants
What does the study involve?
- Patients will be asked to have an annual echocardiogram with right heart pressure assessment (screening for presence of PHT) for four to five years.
- Patients will be asked to obtain a blood test once a year (screening for risk factors) for four to five years.
- Patients will be asked to initiate a treatment with daily “baby” aspirin or low dose Coumadin for three months.
- Patients will be asked to reverse to the drug not taken before (aspirin or low dose Coumadin) for an additional three months.
- Patients will be asked to get a monthly blood test while on these treatments, for a total six months.
Benefits of participating in the study:
- Annual detailed echocardiogram evaluation of right heart pressure (to determine presence of pulmonary hypertension) in addition to the routine echocardiogram review
- Information on the results of laboratory screening for risk factors for developing PHT
- Information on the type of treatment that may better affect and prevent PHT
For more information, please contact Sylvia Titi Singer, MD at Children's Hospital & Research Center at Oakland, 510-428-3169 or ask your clinical care provider.
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