Information

Related Research Units

Gastroenterology, Hepatology and Nutrition Research

Research Overview

Dr. Rufo's current research interests are focused on the development of novel treatments and diagnostic tools for use in the management of children and adults with inflammatory intestinal diseases. Earlier in his career, during his fellowship in the laboratory of Dr. Wayne Lencer, he completed basic research studies that demonstrated that the antifungal antibiotic, clotrimazole, displays both anti-secretory as well as anti-inflammatory properties when studied in vitro and in vivo. As a clinician, Dr. Rufo recognized the potential clinical implication of these findings. He is currently translating these basic science findings into clinical practice, and has obtained FDA-sponsorship for an Orphan Drug Program.

Dr. Rufo and his colleagues are completing a Phase II/III study that is evaluating the efficacy of topical clotrimazole therapy in the treatment of children and adults with pouchitis, an intestinal inflammation found in patients that have undergone colectomy for treatment of ulcerative colitis. This technology has been licensed, and corporate sponsorship has made possible an expansion of this project into a multi-center study to include major academic medical centers. They are currently completing the second stage of the dose escalation study and anticipate that enrollment and data analysis should be completed by the end of the 2007-2008 academic year. If subsequent data support their preliminary findings, topical clotrimazole therapy could have a significant impact on the management of a broad range of inflammatory gastrointestinal disorders, including ulcerative colitis, Crohn disease, and proctitis. A second focus of our translational research program is to develop novel approaches to the diagnosis and interval assessment of patients with inflammatory bowel disease (IBD). They have worked in collaboration with IBD centers at the University of Chicago and at the Mayo Clinic to study the utility of measuring fecal lactoferrin, a white blood cell-derived protein, in patients with IBD. Their published findings demonstrate that FLA levels can help in the assessment of pediatric and adult patients with IBD.

Goals of Dr. Rufo's research include:

  • Investigate and develop new therapies for the treatment of children with Inflammatory Bowel Disease.
  • Develop non-invasive tests to assist in the diagnosis and interval assessment of children with know or suspected Inflammatory Bowel Disease.

Research Background

Dr. Rufo received an MD from the University of Massachusetts Medical School. He received an MMSc degree from Harvard Medical School in 2002 after he completed the Clinical Investigator Training Program sponsored jointly by BIDMC, HMS, and MIT.

Education

Undergraduate School

Harvard University
1985 Cambridge MA

Medical School

University of Massachusetts Medical School
1990 Worcester MA

Internship

Johns Hopkins Hospital
1991 Baltimore MD

Residency

Johns Hopkins Hospital
1993 Baltimore MD

Fellowship

Pediatric Gastroenterology Boston Children's Hospital
1994 Boston MA

Fellowship

Cell Biology Boston Children's Hospital
1996 Boston MA

Fellowship

Pfizer Fellowship in Clinical Investigation Beth Israel Deaconess Medical Center
2002 Boston MA

Media

Caregiver Profile

Meet Dr. Paul Rufo

Publications

  1. Clinical outcomes of maintenance therapy with sulfasalazine compared to 5-aminosalicylates in children with ulcerative colitis. J Pediatr Gastroenterol Nutr. 2025 Apr 10. View Abstract
  2. Higher alpha diversity and Lactobacillus blooms are associated with better engraftment after fecal microbiota transplant in inflammatory bowel disease. Sci Rep. 2024 08 06; 14(1):18188. View Abstract
  3. Evaluation and novel treatment in a case of refractory small bowel-small bowel intussusception. JPGN Rep. 2024 Aug; 5(3):367-370. View Abstract
  4. Inpatient management of iron deficiency anemia in pediatric patients with inflammatory bowel disease: A single center experience. World J Clin Pediatr. 2024 Mar 09; 13(1):89318. View Abstract
  5. Analysis of missed clinic visits, preferred languages, and telemedicine in a pediatric gastroenterology practice. J Pediatr Gastroenterol Nutr. 2024 May; 78(5):1069-1081. View Abstract
  6. Efficacy and Safety of Tacrolimus or Infliximab Therapy in Children and Young Adults With Acute Severe Colitis. J Pediatr Gastroenterol Nutr. 2023 08 01; 77(2):222-227. View Abstract
  7. Advancing Health Equity and Inclusion in an Academic Pediatric Medical Center: Priorities Addressed and Lessons Learned. J Pediatr Soc North Am. 2023 Feb 15; 5(Suppl 1):618. View Abstract
  8. Higher alpha diversity and Lactobacillus blooms are associated with better engraftment after Fecal Microbiota Transplant in Inflammatory Bowel Disease. medRxiv. 2023 Feb 01. View Abstract
  9. Efficacy and Safety of Sulfasalazine Suspension in Children With Ulcerative Colitis. J Pediatr Gastroenterol Nutr. 2023 04 01; 76(4):460-467. View Abstract
  10. The Specific Carbohydrate Diet: Obstacle and Opportunities. JPGN Rep. 2022 Aug; 3(3):e240. View Abstract
  11. Efficacy and Safety of High-dose Cholecalciferol in Patients With Inflammatory Bowel Disease Receiving Infliximab. J Pediatr Gastroenterol Nutr. 2022 04 01; 74(4):476-483. View Abstract
  12. Stratification of risk of progression to colectomy in ulcerative colitis via measured and predicted gene expression. Am J Hum Genet. 2021 09 02; 108(9):1765-1779. View Abstract
  13. Analysis of Using the Total White Blood Cell Count to Define Severe New-onset Ulcerative Colitis in Children. J Pediatr Gastroenterol Nutr. 2020 09; 71(3):354-360. View Abstract
  14. Protein-Losing Enteropathy in the Setting of Iron Deficiency Anemia: A Case Series. JPGN Rep. 2020 Nov; 1(2):e009. View Abstract
  15. Pediatric Collagenous Gastroenterocolitis Successfully Treated with Methotrexate. Case Rep Pediatr. 2020; 2020:1929581. View Abstract
  16. Screening and Counseling for Alcohol Use in Adolescents With Chronic Medical Conditions in the Ambulatory Setting. J Adolesc Health. 2019 06; 64(6):804-806. View Abstract
  17. Clinical and biological predictors of response to standardised paediatric colitis therapy (PROTECT): a multicentre inception cohort study. Lancet. 2019 04 27; 393(10182):1708-1720. View Abstract
  18. Ulcerative colitis mucosal transcriptomes reveal mitochondriopathy and personalized mechanisms underlying disease severity and treatment response. Nat Commun. 2019 01 03; 10(1):38. View Abstract
  19. Effectiveness of oral iron supplementation in treatment of anemia associated with pediatric ulcerative colitis flare. Am J Hematol. 2018 12; 93(12):E404-E406. View Abstract
  20. Compositional and Temporal Changes in the Gut Microbiome of Pediatric Ulcerative Colitis Patients Are Linked to Disease Course. Cell Host Microbe. 2018 10 10; 24(4):600-610.e4. View Abstract
  21. Serologic Reactivity Reflects Clinical Expression of Ulcerative Colitis in Children. Inflamm Bowel Dis. 2018 05 18; 24(6):1335-1343. View Abstract
  22. Commentary: Button Batteries in Fidget Spinners: Is It Time to Push the "Panic Button"? J Pediatr Gastroenterol Nutr. 2018 04; 66(4):557-558. View Abstract
  23. Enhanced Contribution of HLA in Pediatric Onset Ulcerative Colitis. Inflamm Bowel Dis. 2018 03 19; 24(4):829-838. View Abstract
  24. Protein-Losing Enteropathy in the Setting of Severe Iron Deficiency Anemia. J Investig Med High Impact Case Rep. 2018 Jan-Dec; 6:2324709618760078. View Abstract
  25. Histologic Correlates of Clinical and Endoscopic Severity in Children Newly Diagnosed With Ulcerative Colitis. Am J Surg Pathol. 2017 Nov; 41(11):1491-1498. View Abstract
  26. The Role of Environmental Factors in the Pathogenesis of Inflammatory Bowel Diseases: A Review. JAMA Pediatr. 2017 10 01; 171(10):999-1005. View Abstract
  27. A randomized controlled trial of levodopa in patients with Angelman syndrome. Am J Med Genet A. 2018 05; 176(5):1099-1107. View Abstract
  28. Factors associated with early outcomes following standardised therapy in children with ulcerative colitis (PROTECT): a multicentre inception cohort study. Lancet Gastroenterol Hepatol. 2017 12; 2(12):855-868. View Abstract
  29. Correlation of endoscopic disease severity with pediatric ulcerative colitis activity index score in children and young adults with ulcerative colitis. World J Gastroenterol. 2017 May 14; 23(18):3322-3329. View Abstract
  30. Assessment of Fecal ASCA Measurement as a Biomarker of Crohn Disease in Pediatric Patients. J Pediatr Gastroenterol Nutr. 2017 02; 64(2):248-253. View Abstract
  31. A Screening Tool for Assessing Alcohol Use Risk among Medically Vulnerable Youth. PLoS One. 2016; 11(5):e0156240. View Abstract
  32. Health Care Maintenance in Adolescents with Inflammatory Bowel Disease. Adolesc Med State Art Rev. 2016; 27(1):177-92. View Abstract
  33. Electrophysiological Studies into the Safety of the Anti-diarrheal Drug Clotrimazole during Oral Rehydration Therapy. PLoS Negl Trop Dis. 2015 Sep; 9(9):e0004098. View Abstract
  34. Inflammatory bowel disease and neoplasia in children. Dig Dis. 2014; 32(4):455-62. View Abstract
  35. NASPGHAN guidelines for training in pediatric gastroenterology. J Pediatr Gastroenterol Nutr. 2013 Jan; 56 Suppl 1:S1-8. View Abstract
  36. Health supervision in the management of children and adolescents with IBD: NASPGHAN recommendations. J Pediatr Gastroenterol Nutr. 2012 Jul; 55(1):93-108. View Abstract
  37. Desquamative enteropathy and pyloric atresia without skin disease caused by a novel intracellular beta4 integrin mutation. J Pediatr Gastroenterol Nutr. 2008 Nov; 47(5):585-91. View Abstract
  38. Increased incidence of urinary matrix metalloproteinases as predictors of disease in pediatric patients with inflammatory bowel disease. Inflamm Bowel Dis. 2008 Aug; 14(8):1091-6. View Abstract
  39. Adalimumab for the treatment of Crohn-like colitis and enteritis in glycogen storage disease type Ib. J Inherit Metab Dis. 2008 Dec; 31 Suppl 3:505-9. View Abstract
  40. Adalimumab for the treatment of Crohn-like colitis and enteritis in glycogen storage disease type Ib. Journal of Inherited Metababolic Disease. 2008; [Epub ahead of print] . View Abstract
  41. Challenges and progress in pediatric inflammatory bowel disease. Curr Opin Gastroenterol. 2007 Jul; 23(4):406-12. View Abstract
  42. Fecal lactoferrin is a sensitive and specific marker of disease activity in children and young adults with inflammatory bowel disease. J Pediatr Gastroenterol Nutr. 2007 Apr; 44(4):414-22. View Abstract
  43. Pancreatitis as the initial manifestation of stage IV neuroblastoma. J Pediatr Gastroenterol Nutr. 2007 Jan; 44(1):146-8. View Abstract
  44. Why good pouches go bad. Inflamm Bowel Dis. 2007 Jan; 13(1):116-7. View Abstract
  45. Pancreatitis as the intitial manifestation of Stage IV Neuroblastoma - A Case Report. Journal of Pediatric Gastroenterology Hepatology and Nutrition. 2007; 44(1):146-148. View Abstract
  46. Fecal ASCA Measurements in the Assessment of Pediatric Patients with Crohn’s Disease (CD) or Suspected Inflammatory Bowel Disease (IBD). American College of Gastroenterology (Las Vegas, NV). 2006. View Abstract
  47. Current Therapy of Inflammatory Bowel Disease in Children . Pediatric Drugs. 2006; 8(5):279-302. View Abstract
  48. Current therapy of inflammatory bowel disease in children. Paediatr Drugs. 2006; 8(5):279-302. View Abstract
  49. Severe colitis in children. J Pediatr Gastroenterol Nutr. 2005 Oct; 41(4):375-85. View Abstract
  50. IBD in children: the more you look, the more you see.... Inflamm Bowel Dis. 2004 May; 10(3):327-8. View Abstract
  51. Serial Fecal Lactoferrin Measurements are Useful in the Interval Assessment of Patients with Active and Inactive Inflammatory Bowel Disease. Digestive Disease Week - American Gastroenterological Association. 2004. View Abstract
  52. Diarrhea – Pediatric. The Encyclopedia of Gastroenterology. 2004; 1:585-593. View Abstract
  53. The Detection of Lactoferrin, ASCA and ANCA in Feces is useful for Assessing Pediatric IBD Patients. Digestive Disease Week - American Gastroenterological Association. 2004. View Abstract
  54. Anti-inflammatory Activities Displayed by Triphenyl and Imidazole Containing Compounds. 2004. View Abstract
  55. Diarrhea-associated HIV-1 APIs potentiate muscarinic activation of Cl- secretion by T84 cells via prolongation of cytosolic Ca2+ signaling. Am J Physiol Cell Physiol. 2004 May; 286(5):C998-C1008. View Abstract
  56. Fecal lactoferrin is a sensitive and specific marker in identifying intestinal inflammation. Am J Gastroenterol. 2003 Jun; 98(6):1309-14. View Abstract
  57. Enhancement of intracellular Ca Signaling and Chloride Transport by Nelfinavir and Other Aspartyl Protease inhibitors. 2003. View Abstract
  58. Pouchitis: Evolution of a New Form of Inflammatory Bowel Disease. Diarrhea Digest. 2000. View Abstract
  59. The antifungal antibiotic, clotrimazole, inhibits chloride secretion by human intestinal T84 cells via blockade of distinct basolateral K+ conductances. Demonstration of efficacy in intact rabbit colon and in an in vivo mouse model of cholera. J Clin Invest. 1997 Dec 15; 100(12):3111-20. View Abstract
  60. Proteolytic activation of cholera toxin and Escherichia coli labile toxin by entry into host epithelial cells. Signal transduction by a protease-resistant toxin variant. J Biol Chem. 1997 Jun 13; 272(24):15562-8. View Abstract
  61. Proteolytic Activation of cholera toxin by Eschericia coli labile toxin by entry into host epithelial cells: signal transduction by a proteolytic resistant toxin variant. J. Biol. Chem. 1997; 272:15562-15568. View Abstract
  62. The antifungal antibiotic, clotrimazole, inhibits Cl- secretion by polarized monolayers of human colonic epithelial cells. J Clin Invest. 1996 Nov 01; 98(9):2066-75. View Abstract
  63. Transcytosis of cholera toxin subunits across model human intestinal epithelia. Proc Natl Acad Sci U S A. 1995 Oct 24; 92(22):10094-8. View Abstract
  64. Pulmonology. The Harriet Lane Handbook, K.B. Johnson e.d. 1994; 80-94. View Abstract
  65. How families cope with diabetes in adolescence. An approach and case analyses. Pediatrician. 1988; 15(1-2):80-94. View Abstract

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