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Paul Szabolcs, MD

Director, Blood and Marrow Transplantation, Cellular Therapies

Professor of Pediatrics and Immunology at university of Pittsburgh, USA

Biography

I have built and since 2011 lead the Division of Blood and Marrow Transplantation and Cellular Therapies at UPMC Children's Hospital of Pittsburgh (CHP). I am a physician-scientist whose clinical and laboratory research interests focus on understanding tolerance and immune competence after allogeneic transplantation with the overall goal of developing safer and more effective therapies. I have designed and launched novel trials of reduced-intensity conditioning for patients suffering from inborn errors of hematopoiesis ( Thalassemia, DBA, Sickle cell), immune deficiencies and inborn errors of metabolism in children and young adults. The clinical outcomes and immune reconstitution results of our trial https://clinicaltrials.gov/ct2/show/NCT01962415 were reported in Blood Advances: PMID:32634238 where for the first time reduced intensity (RIC) cord blood transplantation offered superior outcomes compared to myeloablative approaches with ~95% overall survival, >92% event free survival for >20 genetic diseases despite comorbidities.
Other innovative protocols address if autologous CD34+ immunoablative but reduced toxicity stem cell rescue can induce remission in refractory autoimmune diseases e.g. systemic sclerosis https://clinicaltrials.gov/ct2/show/NCT03630211.
Since I performed the first ever successful cadaveric BMT after bilateral lung transplant in 2010 procured from the same donor (JACI, 2015:13:2:567-570), I have developed an NIH-funded trial of tandem cadaveric bilateral orthotopic lung transplant and bone marrow transplant (BOLT-BMT). Teenagers and young adults are treated with an underlying immunodeficiency syndrome where BMT is intended to improve immunity while also possibly induce solid organ tolerance,
https://clinicaltrials.gov/ct2/show/NCT03330795. This concept is now tested with liver transplant and other organs in those with underlying immune or bone marrow disease.