Sickle cell disease (SCD) is a chronic hemolytic disorder characterized by recurrent vaso-occlusive episodes (VOC) and progressive organ damage. It strikes at a young age, with a clinical course punctuated by painful disabling crises, yet treatment options are few. Once patients develop VOC, intervention is limited to pain alleviation; no therapy is currently available to alter the course of VOC. Therefore, efforts are needed to evaluate potential therapeutic interventions and determine the potential impact. My research efforts have focused on pediatric and adult patients with SCD, with a specific goal of measuring coagulation by innovative technologies and determining potential therapeutic options. I recently completed a small pilot study evaluating prophylactic dose anticoagulation for patients with SCD admitted for VOC. I found a greater absolute decrease in pain scores and shorter time on patient controlled analgesia, both arguing for further investigations. I now have positioned myself to examine the effect of an oral factor Xa inhibitor administered daily to decrease chronic daily pain and hypercoagulability. Outcomes will include daily pain scores, patient reported outcomes and hypercoagulable markers. To provide a comprehensive evaluation of the impact of pain in patients with SCD, I will use Patient Reported Outcome Measurement Information System (PROMIS), a computerized adaptive questionnaire, to evaluate fatigue, physical function and pain impact. Pain scores will be recorded through the Sickle cell Mobile Application to Record symptoms via Technology (SMART) app. The SMART app was developed by my research group to track symptoms and improve medication adherence. To allow successful completion of the study, I have established solid mentorship with the leaders in coagulation and SCD, in addition to assembling a scientific advisory board to promote further expertise and insight. I will also pursue completion of coursework required to obtain a Masters in Clinical Research, to strengthen my background in clinical research methodology. Support from this award will provide the opportunity for me to gain the necessary experience, didactic training, and mentorship to become a successful and independent researcher in hematology. Importantly, it will also build a framework for my long-term career in academic medicine; performing innovative research evaluating novel therapeutic agents in patients with SCD.
Greater than 50% of patients with SCD have significant daily pain and all patients have increased clotting markers. This proposal will evaluate prophylactic dose anticoagulation using a novel therapeutic drug for SCD to reduce pain and the known hypercoagulable state.
| Jonassaint, Charles R; Kang, Chaeryon; Abrams, Daniel M et al. (2018) Understanding patterns and correlates of daily pain using the Sickle cell disease Mobile Application to Record Symptoms via Technology (SMART). Br J Haematol 183:306-308 |
| Wang, Hsin-Hsiao Scott; Herbst, Katherine W; Rothman, Jennifer A et al. (2016) Trends in Sickle Cell Disease-related Priapism in U.S. Children's Hospitals. Urology 89:118-22 |
