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Janet L Kwiatkowski, MD, MSCE

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Professor of Pediatrics (Hematology) at the Children's Hospital of Philadelphia
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Consulting Hematologist, Hospital of the University of Pennsylvania
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Consulting Hematologist, Pennsylvania Hospital
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Director, Thalassemia Program, Children's Hospital of Philadelphia
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Clinical Director, Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED)
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Department: Pediatrics
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46 Contact information
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The Children's Hospital of Philadelphia
1e Division of Hematology
1e 3500 Civic Center Blvd
32 Hub Room #13547
Philadelphia, PA 19104
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2e Office: 215-590-5286
32 Fax: 215-590-3992
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13 Education:
21 9 B.S. 14 (Zoology) c
39 Duke University, summa cum laude, 1988.
21 9 M.D. c
4e Columbia University, College of Physicians & Surgeons, 1992.
21 9 MSCE 34 (Clinical Epidemiology and Biostatistics) c
33 University of Pennsylvania, 2003.
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Description of Research Expertise

fa Optimization of iron chelation, monitoring transfusional iron overload, stroke prevention in sickle cell disease, headaches in sickle cell disease, complications of thalassemia, transfusion therapy, gene therapy for hemoglobinopathies
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Description of Clinical Expertise

96 General pediatric hematology, iron deficiency anemia, thalassemia, sickle cell disease, transfusional iron overload, chelation therapy
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Selected Publications

18d Gibson NM, Elgarten CW, Oved JH, Wray L, Freedman J, Khandros E, Worster E, Nicholas P, Kadauke S, Wang Y, Grupp S, Kwiatkowski JL, Olson TS: Outcomes of unrelated donor stem cell transplantation with partial T cell depletion in pediatric patients with hemoglobinoopathies. Transplant Cell Therapy 31(10): e1-828, Oct 2025.

e1 Kwiatkowski JL: Iron chelation: choice of agent, dosing, and adverse effects. Up to Date. Elliot P. Vichinsky and Jennifer S. Tirnauer (eds.). May 2025.

141 Gibson NM, Khandros E, Elgarten CW, Worster E, Mono DS, Thompson AA, Kwiatkowski JL, Olson TS: Impact of HLA alloimmunization in gene-modified autologous stem cell transplant for transfusion-dependent thalassemia. Blood 145(22): 2666-70, May 2025.

17b Klaassen RM, Khelifi1 RS, Barrowman N, Merelles-Pulcini M, Vichinsky EP, Kirby-Allen M, Neufeld EJ, Kwiatkowski JL, Wu J, and Young NL: Taking care of the caregivers: A quality of life measure for the parents of children with transfusion-dependent thalassemia. Pediatr Blood & Cancer 2025 Notes: in press.

1e8 de la Fuente J, Frangoul H, Lang P, Wall D, Meisel R, Corbacioglu S, Li AM, Shah AJ, Carpenter B, Kwiatkowski JL, Mapara MUY, Liem RI, Rupprecht J, Kuo KHM, Berkeley H, Algeri M, Smith W, Kohli P, Li N, Rubin J, Zhang S, Hobbs WE, Locatelli F: Improvements in health related quality of life in patients with transfusion-dependent beta-thalassemia after Exagamglogene Autotemcel Blood Advances 2025 Notes: in press.

142 Padarti A, Quinn CT, Kwiatkowski JL, McNeill M, Kirkham FJ, Plumb P, Hynan LS, Dowling MM, PFAST Investigators: Prevalence of antiplatelet and anticoagulation therapy in children with sickle cell anemia and stroke. Pediatr Stroke 11: 111-140, 2025.

f1 Kwiatkowski JL, Walters MC, Hongeng S, Yannaki E, Kulozik AE, Kunz JB, Sauer MG, Thraher AJ, Thuret I, Lal A, Tao G, Ali S, Thakar HL, Elliot H, Lodaya A, Lee J, Colvin RA, Locatelli F, Thompson AA 11a : Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype beta thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial. The Lancet 404(10468): 2175-2186, Nov 2024.

299 Alexis Thompson, Janet L. Kwiatkowski, Jennifer Schneiderma, Isabelle Thuret, Andreas E. Kulozik, Evangelia Yannaki, Marina Cavazzana,Suradej Hongeng, Timothy S. Olson, Martin G. Sauer, Adrian J. Thrasher, Ashutosh Lal, John E. J. Rasko, Shamshad Ali, Ge Tao, Himal L. Thakar, Ami Deora, Clark Paramore, Niki Witthuhn, Mark C. Walters, and Franco Locatelli: Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia. Blood, Abstract 2194 Nov 2024 Notes: Poster presentation.

1b9 Gibson N, Amaral S, Hsu JY, Nguyen N, Haney M, Heimbruch KE, Sabulski A, Davidow KA, Lind K, Scanlon S, Alva H, Bailey LC, Olson TS, Kwiatkowski JL: Defining the Impacts of Iron Overload in Children Receiving Hematopoietic Stem Cell Transplantation for Non-Malignant Hematologic Disorders: A Multicenter Analysis. Blood Abstract 1064 Nov 2024 Notes: Oral presentation.

11c Dain AS, Li Y, Master S, Raffini L, Getz KD, and Kwiatkowski JL: Hydroxyurea Initiation and Occurrence of Cerebrovascular Outcomes in Sickle Cell Disease. Blood Abstract 2504 Nov 2024 Notes: Poster Presentation.

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