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.

About Paul Rufo

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.

Researcher Services

Researcher Areas

  • Inflammatory Bowel Disease

Research Departments

Research Divisions


Publications powered by Harvard Catalyst Profiles

  1. Haberman Y, Karns R, Dexheimer PJ, Schirmer M, Somekh J, Jurickova I, Braun T, Novak E, Bauman L, Collins MH, Mo A, Rosen MJ, Bonkowski E, Gotman N, Marquis A, Nistel M, Rufo PA, Baker SS, Sauer CG, Markowitz J, Pfefferkorn MD, Rosh JR, Boyle BM, Mack DR, Baldassano RN, Shah S, Leleiko NS, Heyman MB, Grifiths AM, Patel AS, Noe JD, Aronow BJ, Kugathasan S, Walters TD, Gibson G, Thomas SD, Mollen K, Shen-Orr S, Huttenhower C, Xavier RJ, Hyams JS, Denson LA. 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
  2. Egberg MD, Mitchell PD, Galanko JA, Rufo PA. Effectiveness of oral iron supplementation in treatment of anemia associated with pediatric ulcerative colitis flare. Am J Hematol. 2018 Dec; 93(12):E404-E406. View abstract
  3. Schirmer M, Denson L, Vlamakis H, Franzosa EA, Thomas S, Gotman NM, Rufo P, Baker SS, Sauer C, Markowitz J, Pfefferkorn M, Oliva-Hemker M, Rosh J, Otley A, Boyle B, Mack D, Baldassano R, Keljo D, LeLeiko N, Heyman M, Griffiths A, Patel AS, Noe J, Kugathasan S, Walters T, Huttenhower C, Hyams J, Xavier RJ. 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
  4. Spencer EA, Davis SM, Mack DR, Boyle BM, Griffiths AM, LeLeiko NS, Sauer CG, Keljo DJ, Markowitz JF, Baker SS, Rosh JR, Baldassano RN, Oliva-Hemker M, Pfefferkorn MD, Otley AR, Heyman MB, Noe JD, Patel AS, Rufo PA, Alison Marquis M, Walters TD, Collins MH, Kugathasan S, Denson LA, Hyams JS, Dubinsky MC. Serologic Reactivity Reflects Clinical Expression of Ulcerative Colitis in Children. Inflamm Bowel Dis. 2018 May 18; 24(6):1335-1343. View abstract
  5. Bousvaros A, Rufo P. Commentary: Button Batteries in Fidget Spinners: Is It Time to Push the "Panic Button"? J Pediatr Gastroenterol Nutr. 2018 Apr; 66(4):557-558. View abstract
  6. Venkateswaran S, Prince J, Cutler DJ, Marigorta UM, Okou DT, Prahalad S, Mack D, Boyle B, Walters T, Griffiths A, Sauer CG, LeLeiko N, Keljo D, Markowitz J, Baker SS, Rosh J, Pfefferkorn M, Heyman MB, Patel A, Otley A, Baldassano R, Noe J, Rufo P, Oliva-Hemker M, Davis S, Zwick ME, Gibson G, Denson LA, Hyams J, Kugathasan S. Enhanced Contribution of HLA in Pediatric Onset Ulcerative Colitis. Inflamm Bowel Dis. 2018 03 19; 24(4):829-838. View abstract
  7. Yasuda JL, Rufo PA. 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
  8. Boyle B, Collins MH, Wang Z, Mack D, Griffiths A, Sauer C, Markowitz J, LeLeiko N, Keljo D, Rosh J, Baker SS, Pfefferkorn M, Heyman M, Patel A, Baldassano R, Noe J, Rufo P, Kugathasan S, Walters T, Denson L, Hyams J. 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
  9. Shouval DS, Rufo PA. 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
  10. Tan WH, Bird LM, Sadhwani A, Barbieri-Welge RL, Skinner SA, Horowitz LT, Bacino CA, Noll LM, Fu C, Hundley RJ, Wink LK, Erickson CA, Barnes GN, Slavotinek A, Jeremy R, Rotenberg A, Kothare SV, Olson HE, Poduri A, Nespeca MP, Chu HC, Willen JM, Haas KF, Weeber EJ, Rufo PA. A randomized controlled trial of levodopa in patients with Angelman syndrome. Am J Med Genet A. 2018 05; 176(5):1099-1107. View abstract
  11. Hyams JS, Davis S, Mack DR, Boyle B, Griffiths AM, LeLeiko NS, Sauer CG, Keljo DJ, Markowitz J, Baker SS, Rosh J, Baldassano RN, Patel A, Pfefferkorn M, Otley A, Heyman M, Noe J, Oliva-Hemker M, Rufo P, Strople J, Ziring D, Guthery SL, Sudel B, Benkov K, Wali P, Moulton D, Evans J, Kappelman MD, Marquis A, Sylvester FA, Collins MH, Venkateswaran S, Dubinsky M, Tangpricha V, Spada KL, Britt A, Saul B, Gotman N, Wang J, Serrano J, Kugathasan S, Walters T, Denson LA. 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
  12. Kerur B, Litman HJ, Stern JB, Weber S, Lightdale JR, Rufo PA, Bousvaros A. 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
  13. Tang V, Valim C, Moman R, Richman A, Zhou J, Ramgopal V, Albert R, Boone JH, Rufo PA. 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
  14. Levy S, Dedeoglu F, Gaffin JM, Garvey KC, Harstad E, MacGinnitie A, Rufo PA, Huang Q, Ziemnik RE, Wisk LE, Weitzman ER. A Screening Tool for Assessing Alcohol Use Risk among Medically Vulnerable Youth. PLoS One. 2016; 11(5):e0156240. View abstract
  15. Rufo PA, Egberg MD. Health Care Maintenance in Adolescents with Inflammatory Bowel Disease. Adolesc Med State Art Rev. 2016; 27(1):177-92. View abstract
  16. Lexmond WS, Rufo PA, Fiebiger E, Lencer WI. 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
  17. Rufo PA. Inflammatory bowel disease and neoplasia in children. Dig Dis. 2014; 32(4):455-62. View abstract
  18. Leichtner AM, Gillis LA, Gupta S, Heubi J, Kay M, Narkewicz MR, Rider EA, Rufo PA, Sferra TJ, Teitelbaum J. NASPGHAN guidelines for training in pediatric gastroenterology. J Pediatr Gastroenterol Nutr. 2013 Jan; 56 Suppl 1:S1-8. View abstract
  19. Rufo PA, Denson LA, Sylvester FA, Szigethy E, Sathya P, Lu Y, Wahbeh GT, Sena LM, Faubion WA. 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
  20. Salvestrini C, McGrath JA, Ozoemena L, Husain K, Buhamrah E, Sabery N, Leichtner A, Rufo PA, Perez-Atayde A, Orteu CH, Torrente F, Heuschkel RB, Thomson MA, Murch SH. 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
  21. Manfredi MA, Zurakowski D, Rufo PA, Walker TR, Fox VL, Moses MA. 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
  22. Davis MK, Rufo PA, Polyak SF, Weinstein DA. 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
  23. Davis, MK Rufo, PA Polyak, SF Weinstein, DA. 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
  24. Rufo PA, Bousvaros A. Challenges and progress in pediatric inflammatory bowel disease. Curr Opin Gastroenterol. 2007 Jul; 23(4):406-12. View abstract
  25. Walker TR, Land ML, Kartashov A, Saslowsky TM, Lyerly DM, Boone JH, Rufo PA. 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
  26. Fishman DS, Manfredi MA, LaBelle JL, Levine JE, Shamberger RC, Rufo PA. Pancreatitis as the initial manifestation of stage IV neuroblastoma. J Pediatr Gastroenterol Nutr. 2007 Jan; 44(1):146-8. View abstract
  27. Hait E, Rufo PA. Why good pouches go bad. Inflamm Bowel Dis. 2007 Jan; 13(1):116-7. View abstract
  28. Fishman DS, Manfredi M, LaBelle J, Levine J, Shamberger RS, Rufo PA. 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
  29. Rufo PA, Bousvaros A. Current therapy of inflammatory bowel disease in children. Paediatr Drugs. 2006; 8(5):279-302. View abstract
  30. Rufo, PA, Bousvaros, AB. Current Therapy of Inflammatory Bowel Disease in Children . Pediatric Drugs. 2006; 8(5):279-302. View abstract
  31. Vivian Tang, Thomas R. Walker, Tracee Saslowsky, Kimberly Ong, Lori Rosenberg, James Boone, Krista Tygrett, Paul A. Rufo . 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
  32. Kugathasan S, Dubinsky MC, Keljo D, Moyer MS, Rufo PA, Wyllie R, Zachos M, Hyams J. Severe colitis in children. J Pediatr Gastroenterol Nutr. 2005 Oct; 41(4):375-85. View abstract
  33. Rufo PA. IBD in children: the more you look, the more you see.... Inflamm Bowel Dis. 2004 May; 10(3):327-8. View abstract
  34. Thomas R Walker, Michelle L. Land, Tracee M. Cook, William Sandborn, Therese Johnson, James Boone, David Lyerly, Paul A. Rufo. 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
  35. Rufo, P.A. Lencer, W.I. Anti-inflammatory Activities Displayed by Triphenyl and Imidazole Containing Compounds. 2004. View abstract
  36. James Boone, Darcy Whitlock, Daniel Sisco, Thomas Walker, Paul Rufo . 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
  37. Rudolph, J.A and Rufo, P.A. Diarrhea – Pediatric. The Encyclopedia of Gastroenterology. 2004; 1:585-593. View abstract
  38. Rufo PA, Lin PW, Andrade A, Jiang L, Rameh L, Flexner C, Alper SL, Lencer WI. 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
  39. Kane SV, Sandborn WJ, Rufo PA, Zholudev A, Boone J, Lyerly D, Camilleri M, Hanauer SB. Fecal lactoferrin is a sensitive and specific marker in identifying intestinal inflammation. Am J Gastroenterol. 2003 Jun; 98(6):1309-14. View abstract
  40. Lencer, W.I Alper, S.A. Lin, P. Rufo, P.A. Enhancement of intracellular Ca Signaling and Chloride Transport by Nelfinavir and Other Aspartyl Protease inhibitors. 2003. View abstract
  41. Rufo, P.A. Pouchitis: Evolution of a New Form of Inflammatory Bowel Disease. Diarrhea Digest. 2000. View abstract
  42. Rufo PA, Merlin D, Riegler M, Ferguson-Maltzman MH, Dickinson BL, Brugnara C, Alper SL, Lencer WI. 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
  43. Lencer WI, Constable C, Moe S, Rufo PA, Wolf A, Jobling MG, Ruston SP, Madara JL, Holmes RK, Hirst TR. 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
  44. Lencer, W.I., Constable, C., Moe, S., Rufo, P.A., Wolf, A. Jobling, M.G. Webb, H.M., Ruston, S., Madara, J.L., Holmes, R.K. 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
  45. Rufo PA, Jiang L, Moe SJ, Brugnara C, Alper SL, Lencer WI. 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
  46. Lencer WI, Moe S, Rufo PA, Madara JL. 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
  47. Rufo, P.A. Pulmonology. The Harriet Lane Handbook, K.B. Johnson e.d. 1994; 80-94. View abstract
  48. Hauser ST, Paul EL, Jacobson AM, Weiss-Perry B, Vieyra MA, Rufo P, Spetter LD, DiPlacido J, Wertlieb D, Wolfsdorf J, et al. How families cope with diabetes in adolescence. An approach and case analyses. Pediatrician. 1988; 15(1-2):80-94. View abstract