New Insights into the Development and Treatment of Immune Thrombocytopenia
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CitationAyad, Nardeen. 2022. New Insights into the Development and Treatment of Immune Thrombocytopenia. Master's thesis, Harvard Medical School.
AbstractImmune Thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by a low platelet count resulting from platelet destruction and impaired platelet production. The incidence of ITP is estimated to be 2 to 5 per 100,000 persons in the general population. The disease affects the quality of life, and bleeding tendency is associated with higher risk of morbidity and mortality among these patients. There are several unanswered questions with regards to the development and management of immune thrombocytopenia. Persistent isolated mild thrombocytopenia that does not meet the American Society of Hematology criteria for ITP (platelet count between 100 and 149 × 10^9/L) remains a concern of uncertain clinical significance among healthcare practitioners and common reason for hematology referral. There are no practice guidelines to direct workup or management of these patients due to the scarcity of data about their prognosis. Therefore, our study investigated the likelihood of development of defined hematologic pathology over up to 32 years of follow up of these otherwise healthy cohort of individuals. The second controversial topic that we choose to study in this project is the treatment choice for children with chronic ITP. In the 30% of children with immune thrombocytopenia who experience a disease course longer than 12 months, little evidence exists comparing the response to first-line treatment options, namely the thrombopoietin receptor agonists and rituximab. Up to date, there is wide variation in the results reported by prospective clinical trials in this field and the treatment choice is dependent on the patient/parent preference, route of administration, side effects, adherence to treatment, and possibility of remission.
Citable link to this pagehttps://nrs.harvard.edu/URN-3:HUL.INSTREPOS:37371560