Case Study: The Thalassemia Clinical Research Network Research
| At a Glance |
Research Question
What are the most effective treatment regimens in the treatment and prevention of Thalassemia complications?
NERI's Role
The TCRN contributed to the advancement of public health for Thalassemia patients.
Results
By virtue of conducting 2 longitudinal registries, 4 observational studies, and 4 interventional studies, NERI has contributed to the body of literature for this orphan disease population.
Funding Institution
These studies are supported by the Thalassemia Clinical Research Network Cooperative; the Sickle Cell Disease Clinical Research Network Cooperative; and the National Heart, Lung and Blood Institute.
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Thalassemia and sickle cell disease can shorten life span and impair quality of life. The Clinical Research group is currently conducting several international trials aimed at improving disease management for the millions of people around the world who are affected by these diseases.
NERI's Role
Four studies conducted by NERI for the Thalassemia Clinical Research Network will contribute to the scientific body of knowledge for this set of diseases:
- Thalassemia Longitudinal Cohort Registry will provide a long-term understanding of standards of care, quality of life, and detailed genotypic and phenotypic characterizations.
- Low Bone Mass Observational study in Thalassemia will estimate the prevalence of low bone mass in thalassemia patients and will evaluate the interaction of factors such as endocrine and genetic influences on bone metabolism in hopes of identifying preventive strategies and effective treatments.
- Pulmonary Hypertension in Thalassemia Pilot Interventional Drug Study with Comparison to Controls will evaluate the safety and efficacy of sildenafil to treat pulmonary hypertension (PHT). Thalassemia patients frequently have undetected PHT, and this study will compare how thalassemia patients and those without PHT respond to treatment.
- Pain in Thalassemia Descriptive Studies will assess, using a survey, the prevalence of pain in subjects with transfusion and non-transfusion dependent thalassemia. The Pain in Transfusion sub-study will assess whether reports of pain vary over the transfusion cycle.
NERI also serves as the data coordinating center for two studies of sickle cell disease:
- The IMPROVE trial is investigating the use of patient-controlled analgesia for patients with sickle cell disease who are admitted to a hospital for a pain crisis.
- The PROACTIVE feasibility study will determine if it is possible to design and complete a randomized trial to assess the effectiveness of a transfusion in preventing a pneumonia-like disorder called acute chest syndrome.
Results
Because of its involvement in many studies of thalassemia and sickle cell disease over the past two decades, NERI has become a world-leader in both fundamental and clinical research into these diseases, and is playing a pivotal role in the effort to increase quality of life and life expectancy in these populations.