Osteopenia and/or Osteoporosis (OOS) is becoming an increasingly prevalent chronic disease among Beta Thalassemia Major (BTM) patients, especially now that life expectancy in these patients has considerably improved through regular blood transfusions and iron chelation therapy. With several, complex genetic and acquired factors involved in its pathogenesis, coupled with the heterogeneity in the clinical response of BTM patients to different pharmacological agents, OOS has proven to be particularly difficult to treat. The great majority of treatment options currently available are not curative, but instead are aimed towards managing the symptoms and progression of the disease in patients. General preventative measures, such as iron chelation therapy and hormonal replacement therapy (HRT), are instrumental aspects of the treatment plan; however, the incredible complexity of OOS necessitates an individualized, multidisciplinary approach to management, with a principal therapy that is safe and effective in patients, and that is accompanied by these other supportive measures. This review, through a comprehensive analysis of current literature, includes data from randomized, placebo-controlled trials, double blind and observational clinical studies, and suggests optimal therapeutic interventions for first-line management of OOS. It also addresses treatment options for BTM patients in whom resistance to the recommended first-line therapy develops, or who display secondary endocrine conditions contributing to OOS. In addition to providing a current synopsis of OOS management and the potential of emerging treatment options, this analysis highlights some of the limitations of traditional therapies. In this way, the paper effectively illustrates the current status of TM-induced OOS; it describes what is or isn’t working, as well as underscores the diagnostic and therapeutic challenges continually faced by patients, researchers and clinicians.
| Identifer | oai:union.ndltd.org:bu.edu/oai:open.bu.edu:2144/19171 |
| Date | 03 November 2016 |
| Creators | Khullar, Natasha |
| Source Sets | Boston University |
| Language | en_US |
| Detected Language | English |
| Type | Thesis/Dissertation |
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