As a physician scientist and clinical innovator in pediatric hematopoietic cellular therapy (HCT), my primary focus of investigation is transplant associated thrombotic microangiopathy (TA-TMA), a complication of HCT that is both common and life threatening. Utilizing multiple research methods, my overarching goal of my investigation is to improve mortality in children with TA-TMA by facilitating prompt diagnosis and investigating new therapies. I recently published a series of studies in a large pediatric cohort reporting a high prevalence of this complication after both autologous and allogeneic HCT and associated increased transplant related mortality. I am implementing a clinical tool to improve prompt diagnosis and directly impact clinical care and leading a pilot study of novel blood and imaging biomarkers of TA-TMA.
- Identifying novel blood and imaging biomarkers of TA-TMA that aid in diagnosis, prognosis and detection of subclinical disease
- A prospective cohort study of TA-TMA after implementation of screening guidelines identifying risk factors for developing TA-TMA and increased transplant related mortality
- Determining the cost associated with TA-TMA and the cost effectiveness of Eculizumab
- Novel interventional therapeutic agents for TA-TMA
Schoettler M, Lehmann L, Margossian S, Lee M, Kean LS, Kao P, Ma C, Duncan CN; Risk factors for transplant-associated thrombotic microangiopathy and mortality in a pediatric cohort. Blood Adv 2020; 4 (11): 2536–2547. doi: https://doi.org/10.1182/bloodadvances.2019001242
Halyabar O, Chang MH, Schoettler ML, et al. Calm in the midst of cytokine storm: a collaborative approach to the diagnosis and treatment of hemophagocytic lymphohistiocytosis and macrophage activation syndrome. Pediatr Rheumatol Online J. 2019;17(1):7. Published 2019 Feb 14. doi:10.1186/s12969-019-0309-6
Schoettler M, Duncan C, Lehmann L, Furutani E, Subramaniam M, Margossian S. Ruxolitinib is an effective steroid sparing agent in children with steroid refractory/dependent bronchiolitis obliterans syndrome after allogenic hematopoietic cell transplantation. Bone Marrow Transplant. 2019;54(7):1158‐1160. doi:10.1038/s41409-019-0450-3
Schoettler, M, Duncan C, Lehmann L. Severe, persistent neurotoxicity after transplant associated thrombotic microangiopathy (TA-TMA) in a pediatric patient despite treatment with eculizumab. Pediatr Transplant. 2019; In press.
Schoettler M, Lehmann L, Li A, Ma C, Duncan C. Thrombotic Microangiopathy Following Pediatric Autologous Hematopoietic Cell Transplantation: A Report of Significant End-Organ Dysfunction in Eculizumab-Treated Survivors. Biol Blood Marrow Transplant. 2019;25(5):e163‐e168. doi:10.1016/j.bbmt.2018.12.840
Schoettler ML, Nathan DG. The Pathophysiology of Acquired Aplastic Anemia: Current Concepts Revisited. Hematol Oncol Clin North Am. 2018;32(4):581‐594. doi:10.1016/j.hoc.2018.03.001
Schoettler ML, Graham D, Tao W, et al. Increasing observation rates in low-risk pediatric immune thrombocytopenia using a standardized clinical assessment and management plan (SCAMP® ). Pediatr Blood Cancer. 2017;64(5):10.1002/pbc.26303. doi:10.1002/pbc.26303