Nikhil Pai, BSc, MD, CNSC, FRCPC, FAAP

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Associate Professor of Pediatrics (Gastroenterology, Hepatology and Nutrition) at the Children's Hospital of Philadelphia
Member, Research Institute, Children's Hospital of Philadelphia
Member, Metabolism, Nutrition and Physical Development Research Affinity Group, Children's Hospital of Philadelphia
Member, Institute for Translational Medicine and Therapeutics, University of Pennsylvania School of Medicine
Director, Pediatric Donor Fecal Microbiota Transplant Program, Children's Hospital of Philadelphia
Director, Clinical Trials Division, Center for Microbial Medicine, Children's Hospital of Philadelphia
Department: Pediatrics

Contact information
Children's Hospital of Philadelphia
Division of Gastroenterology, Hepatology and Nutrition
Roberts Center for Pediatric Research
2716 South Street, Room 14321
Philadelphia, PA 19146
Office: 215-590-2997
Fax: 215-590-2799
Education:
BSc (Biology (Distinction),)
University of Western Ontario, 2004.
MD (Medicine,)
McMaster University (Michael G. DeGroote School of Medicine), 2007.
Residency (Pediatrics,)
Hospital for Sick Children (University of Toronto), 2011.
Fellowship (Pediatric Gastroenterology, Hepatology & Nutrition,)
Boston Children's Hospital (Harvard Medical School), 2014.
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Description of Clinical Expertise

I am a double board-certified pediatric gastroenterologist, certified in both general pediatrics and pediatric gastroenterology by the Royal College of Physicians and Surgeons of Canada (RCPSC) and the American Board of Pediatrics (ABP). I am also a Certified Nutrition Support Clinician (CNSC) and a Fellow of the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN-F).

My clinical expertise spans pediatric intestinal failure, complex nutritional disorders, inflammatory bowel disease (IBD), and metabolic liver disease, including pediatric MASLD (metabolic dysfunction-associated steatotic liver disease). I perform both diagnostic and therapeutic pediatric endoscopy and have formal training in the use of FibroScan® for non-invasive liver stiffness assessment. I provide comprehensive enteral and parenteral nutrition support, including advanced nutrition care in hospitalized and medically complex children.

I completed my pediatric gastroenterology fellowship in the physician-scientist track at Boston Children’s Hospital and additional training in pediatric obesity medicine through the Obesity, Metabolism and Nutrition Institute (OMNI) at Massachusetts General Hospital.

During my 9.5 years as an attending pediatric gastroenterologist at McMaster Children’s Hospital in Hamilton, Ontario, I provided broad-based pediatric GI care across the spectrum of gastrointestinal diseases. I served as the medical lead of the hospital’s Intestinal Rehabilitation Program and helped build the institution’s Pediatric Nutrition Service through combined administrative leadership and clinical service delivery. My clinical focus included pediatric nutrition, intestinal failure, and advanced endoscopic care.

At the Children's Hospital of Philadelphia (CHOP), I currently practice in both inpatient and outpatient settings within the Division of Pediatric Gastroenterology, Hepatology & Nutrition. My clinical responsibilities include general pediatric GI, endoscopy, nutrition support, MASLD, and intestinal rehabilitation. I also lead the development of CHOP’s pediatric donor stool bank—the first of its kind in the United States—supporting clinical and investigational use of fecal microbiota transplantation (FMT).

I am committed to delivering multidisciplinary, evidence-based care to children with complex gastrointestinal and nutritional conditions, integrating clinical excellence with a strong foundation in translational research and innovation.

Description of Research Expertise

Dr. Nikhil Pai is a pediatric gastroenterologist and clinician-scientist at the Children's Hospital of Philadelphia (CHOP), and Director of the Clinical Trials Division for the CHOP Center for Microbial Medicine. He leads a translational research program focused on microbiome-host interactions, metabolic liver disease, and novel microbiome-based therapeutics for chronic gastrointestinal disorders in children. His work combines clinical trials and multi-omics diagnostics - including urine metabolomics, radiologic imaging, and serum proteomics - to help discover novel biomarkers and therapeutic targets in pediatric metabolic dysfunction-associated steatotic liver disease (MASLD), inflammatory bowel disease (IBD), and related conditions.

Dr. Pai developed Canada’s first pediatric donor fecal microbiota transplantation (FMT) stool bank and is now leading the development of CHOP’s pediatric FMT stool bank—the first of its kind in the United States. His research has received support through regional and national funding agencies, with over $23 million in grant support to date. He has received multiple awards recognizing his contributions to pediatric and microbiome research, including the American Society for Parenteral and Enteral Nutrition (ASPEN) Stanley J. Dudrick Mid-Career Research Scholar Award, Canadia Nutrition Society (CNS) Young Investigator Award, and holds various academic and research distinctions from the Canadian Institutes of Health Research (CIHR), the American Gastroenterological Association (AGA), and North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN).

Through cross-disciplinary collaborations, Dr. Pai's work focuses on advancing microbiome-based therapeutics, precision nutrition strategies, and biomarker discovery to improve outcomes for children with chronic gastrointestinal and nutritional disorders.

Selected Publications

Pai N, Popov J, Hill L, Hartung E, Grzywacz K, Moayyedi P; McMaster Pediatric Fecal Microbiota Transplant Research Collaboration.: Results of the first pilot randomized controlled trial of fecal microbiota transplant in pediatric ulcerative colitis: Lessons, limitations, and future prospects. Gastroenterology 161(2): 388-393.e3, Aug 2021.

Popov J, Hartung E, Hill L, Chauhan U, Pai N.: Pediatric patient and parent perceptions of fecal microbiota transplantation for the treatment of ulcerative colitis. J Pediatr Gastroenterol Nutr 73(6): 684-688, Dec 2021.

Yamamoto M, Shanmuganathan M, Hart L, Pai N, Britz-McKibbin P: Urinary metabolites enable differential diagnosis and therapeutic monitoring of pediatric inflammatory bowel disease. Metabolites 11(4): 245, Apr 2021.

Lemay JA, Yamamoto M, Kroezen Z, Shanmuganathan M, Ly R, Hart L, Pai N, Britz-McKibbin P.: Lyophilized fecal short-chain fatty acid and electrolyte determination by capillary electrophoresis with indirect UV detection for assessment of pediatric inflammatory bowel disease. J Pharm Biomed Anal 192: 113658, Jan 2021.

Popov J, Caputi V, Nandeesha N, Rodriguez DA, Pai N.: Microbiota-immune interactions in ulcerative colitis and colitis associated cancer and emerging microbiota-based therapies. Int J Mol Sci 22(21): 11365, Oct 2021.

Pai N, Popov J, Hill L, Hartung E.: Protocol for a double-blind, randomised, placebo-controlled pilot study for assessing the feasibility and efficacy of faecal microbiota transplant in a paediatric Crohn's disease population: PediCRaFT Trial. BMJ Open 9(11): e030120, Nov 2019.

Quirt J, Belza C, Pai N, Clause RF, Markovic F, Wong-Sterling S, Avitzur Y, Wales PW.: Reduction of central line-associated bloodstream infections and line occlusions in pediatric intestinal failure patients receiving long-term parenteral nutrition using an alternative locking solution, 4% Tetrasodium ethylenediamine-tetraacetic acid. JPEN J Parenter Enteral Nutr 45(6): 1286-92, Aug 2021.

Nieuwdorp M, Gilijamse PW, Pai N, Kaplan LM.: Role of the microbiome in energy regulation and metabolism. Gastroenterology 146(6): 1525-33, May 2014.

Wang AY, Popov J, Pai N.: Fecal microbial transplant for the treatment of pediatric inflammatory bowel disease. World J Gastroenterol 22(47): 10304-10315, Dec 2016.

Pai N, Margolis KG: The microbiome in early biomarker discovery for neurodevelopmental disorders: A novel research roadmap? Gastroenterology(4), 813-4, Aug 2024.

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Last updated: 05/14/2025
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