Janet L Kwiatkowski, MD, MSCE
Professor of Pediatrics (Hematology) at the Children's Hospital of Philadelphia
Consulting Hematologist, Hospital of the University of Pennsylvania
Consulting Hematologist, Pennsylvania Hospital
Director, Thalassemia Program, Children's Hospital of Philadelphia
Clinical Director, Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED)
Department: Pediatrics
Contact information
The Children's Hospital of Philadelphia
Division of Hematology
3500 Civic Center Blvd
Hub Room #13547
Philadelphia, PA 19104
Division of Hematology
3500 Civic Center Blvd
Hub Room #13547
Philadelphia, PA 19104
Office: 215-590-5286
Fax: 215-590-3992
Fax: 215-590-3992
Email:
kwiatkowski@chop.edu
kwiatkowski@chop.edu
Education:
B.S. (Zoology)
Duke University, summa cum laude, 1988.
M.D.
Columbia University, College of Physicians & Surgeons, 1992.
MSCE (Clinical Epidemiology and Biostatistics)
University of Pennsylvania, 2003.
Permanent linkB.S. (Zoology)
Duke University, summa cum laude, 1988.
M.D.
Columbia University, College of Physicians & Surgeons, 1992.
MSCE (Clinical Epidemiology and Biostatistics)
University of Pennsylvania, 2003.
Description of Research Expertise
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 hemoglobinopathiesDescription of Clinical Expertise
General pediatric hematology, iron deficiency anemia, thalassemia, sickle cell disease, transfusional iron overload, chelation therapySelected Publications
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 : 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.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.
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.
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.
Kwiatkowski JL,Thompson AA, Tricta F, Temin NT, Rozova A, Fradette, C, Badawy, SM : Real-world evidence:Long-term safety of deferiprone in a large cohort of patients with sickle cell disease enrolled in a registry for up to 10 years. Am J Hematol 99(6): 1031-1039, June 2024.
Locatelli F, Lang P, Wall D, Meisel R.,Corbacioglu, S, Li AM, de la Fuente J,Shah AJ, Carpenter B, Kwiatkowski JL, Makpara M, Liam RI, Cappellini MD, Algeri M, Kattamis A, Sheth S, Grupp S, Handgretinger R., Kohli P, Shi D, Ross L, Bobruff Y, Simard C, Xhang L, Morrow PK, Hobbs WE, Frangoul H, CLIMB THAL-111 Study Group: Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia. N Engl J Med 390(18): 1663-1676, May 2024.
Al-Samkari H, Shehata N, Lang-Robertson K, Bianchi P, Glenthoj A, Sheth, S, Neufeld, EJ, Rees, DC, Chonat, S., Kuo, KHM, Rothman, JA, Barcellini, W, van Beers, EJ, Pospisilova, D, Shah, AJ, van Wijk, R, Glader, B, Manu Pereira, MDM, Andres, O, Kallfa, TA, Eber, SW, Gallagher, PG, Kwiatkowski, JL, Galacteros, F., Lander, C, Watson, A, Elbard, R, Peereboom, D, Grace, RF: Diagnosis and management of pyruvate kinase deficiency: international expert guidelines. Lancet Haematol 11(3): e228-e239, March 2024.
Hamdy M, El-Beshlawy A, Verissimo MPA, Kanter J, Inusa B, Williams S, Lee D, Temin NT, Fradette C, Tricta F, Ebeid FSE, Kwiatkowski JL* Elalfy MS* (*Authors contributed equally to the work): Deferiprone versus deferoxamine for transfusional iron overload in sickle cell disease and other anemias: Pediatric subgroup analysis of the randomized, open-label FIRST study. Pediatric Blood & Cancer 71(1): e30711, Jan 2024 Notes: Online ahead of print.
Janet L. Kwiatkowski, Alexis A. Thompson, Fernando Tricta, Noemi Toiber Temin, Anna Rozova, Caroline Fradette, Sherif M. Badawy: Long-term Safety of Deferiprone in Pediatric Patients With Sickle Cell Disease Enrolled in a US Registry. Proceedings of the American Society of Pediatric Hematology Oncology 2024.
Kwiatkowski, JL and Smith-Whitley, K: Hemoglobinopathies. Nelson Textbook of Pediatrics. Kliegman, RM and St. Geme, JW (eds.). Elsevier, 22nd edition, 2024 Notes: In press.