Overview
Bone Marrow Failure Syndromes
General Hematology
Current Appointments & Affiliations
Professor of Pediatrics
·
2022 - Present
Pediatrics, Hematology-Oncology,
Pediatrics
Professor in Medicine
·
2022 - Present
Medicine, Hematology,
Medicine
Recent Publications
Emapalumab use in malignancy-associated hemophagocytic lymphohistiocytosis in the United States: the REAL-HLH study.
Journal Article Blood Adv · March 10, 2026 Malignancy-associated hemophagocytic lymphohistiocytosis (mHLH), a hyperinflammatory syndrome, has poor prognosis and no standard therapy. Emapalumab, a fully human monoclonal antibody that neutralizes the proinflammatory cytokine interferon gamma, is appr ... Full text Link to item CiteEltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment: The PINES Randomized Clinical Trial.
Journal Article JAMA · November 25, 2025 IMPORTANCE: Eltrombopag, a thrombopoietin receptor agonist, is approved by the US Food and Drug Administration for children with chronic immune thrombocytopenia. Efficacy of eltrombopag during the newly diagnosed phase of pediatric immune thrombocytopenia ... Full text Link to item CiteEnd-of-study results from the ICON3 pines trial, a phase 3, randomized trial of eltrombopag vs. standard first-line treatment for newly diagnosed immune thrombocytopenia in children
Conference Blood · November 3, 2025 AbstractBackground: The Pediatric ITP Newly diagnosed patients Eltrombopag vs Standard therapy (PINES) trial, NCT03939637, was an investiga ... Full text CiteRecent Grants
A global phase 3, randomised, double-blind and placebo-controlled study evaluating the efficacy and safety of etavopivat in adolescents and adults with sickle cell disease
Clinical TrialPrincipal Investigator · Awarded by Novo Nordisk, Inc. · 2025 - 2027PROTOCOL AG348-C-008 Pyruvate Kinase Deficiency Global Longitudinal Registry
Clinical TrialPrincipal Investigator · Awarded by Agios Pharmaceuticals, Inc. · 2018 - 2027AG348-C-023
Clinical TrialPrincipal Investigator · Awarded by Agios Pharmaceuticals, Inc. · 2022 - 2027View All Grants
Education, Training & Certifications
University of Medicine and Dentistry of New Jersey ·
2003
M.D.