Chronic Graft-versus-Host Disease in the Oral Cavity of Patients Following Allogeneic Hematopoietic Stem Cell Transplant and Including Healthy Controls

NCT03602599 | Recruiting

• 2 other identifiers: 18012118-D-0121
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 1

Brief Summary

Background: This study follows people who have had, or will soon have, a transplant using stem cells from another person. This is known as an allogeneic hematopoietic stem cell transplant (HSCT). Graft-versus-host disease (cGVHD) can happen after HSCT. cGVHD can cause mouth problems and more serious issues. Researchers want to study changes in the mouth that might indicate cGVHD. Objective: To identify cGVHD in the mouth and better understand the development, treatment, and progress of post-transplant changes in the mouth. Eligibility: Adults at least 18 years old who will soon undergo HSCT or have had one in the past 3 years Healthy adults at least 18 years old Design: All participants will have a screening visit and baseline visit. They will last 60-90 minutes. Over these two visits, participants will have: Medical and dental history Dental exam. Questions about their eating habits and general health Blood drawn through a needle in the arm Vital signs taken Pictures of their mouth and lips taken Questions about their oral health, including about pain, sensitivity, or dryness Saliva samples taken. Participants will spit into a sterile plastic tube. Swabs taken of the mouth and some of the saliva, plaque, and fluid from the spaces between teeth and gums. Participants may also have: A piece of skin taken (biopsy) from the inner lining of the cheeks A piece of skin taken (biopsy) from the lower lip Dental X-rays Urine pregnancy test Most participants will have at least 7 study visits over 3 years. They will meet with a dentist and repeat baseline tests.

Conditions

Graft Versus Host Disease

Keywords

Mouth; Alloimmunity; Autoimmunity; Salivary Gland; Ulcer; Natural History

Outcome Measures

Primary Outcomes (1)

• Advance understanding of chronic graft versus host disease (cGVHD) pathophysiology and generate hypotheses for future cGVHD studies by tracking the longitudinal development of cGVHD in the oral cavity.

  Advance understanding of chronic graft versus host disease (cGVHD) pathophysiology and generate hypotheses for future cGVHD studies by tracking the longitudinal development of cGVHD in the oral cavity.

  3 years from enrollment of last participant

Secondary Outcomes (1)

• Characterize post-transplant changes in oral cavity in presence and absence of cGVHD to develop better protocols for supportive dental care, Refine clinical definitions and classifications of oral cGVHD, Characterize longitudinal changes, includ...

  3 years after enrollment of last participant

Study Arms (4)

Healthy-controls Longitudinal Cohort

( Cohort HL ; approximate n=20) includes subjects who will participate in up to 4 study visits across 1 year.

Healthy-controls Short-term Cohort

( Cohort HS ; approximate n=80) will participate in a single baseline visit.

New Transplant Cohort

( Cohort NT ; approximate n=300) consists of patients who are scheduled to undergo allogeneic HSCT (under another protocol at the NIH).

Prior Transplant Cohort

( Cohort PT ; approximate n=100) consists of patients who have already undergone allogeneic HSCT.

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Subjects in Cohort NT and Cohort PT will be scheduled to undergo allogeneic hematopoietic stem cell transplant at the NIH (Cohort NT) or have already undergone allogeneic HSCT at the NIH or another institution (Cohort PT) and are within 3 years of most recent bone marrow transplant. Subjects in Cohort HL and Cohort HS will be healthy volunteers and may potentially be age- and/or sex-matched with a transplant patient participant. All subjects will be at least 18 years of age.

You may qualify if:

• Subjects in Cohort NT and Cohort PT must satisfy all of the following criteria in order to participate in the study:
• Scheduled to undergo allogeneic HSCT at the NIH (Cohort NT) or have already undergone allogeneic HSCT at the NIH or another institution (Cohort PT) and are within 3 years of most recent bone marrow transplant
• Willing to participate in protocol evaluations including saliva collection, oral swab collection, and completion of patient-reported outcome (PRO) forms
• Willing to return to the NIH Dental Clinic for scheduled evaluations
• Be at least 18 years of age at screening
• Be able to understand and provide written informed consent
• Subjects in Cohort HL and Cohort HS must satisfy all of the following criteria in order to participate in the study:
• Willing to undergo oral exam and participate in Visit 1 protocol evaluations including saliva collection, oral swab collection and oral biopsies.
• Be at least 18 years of age at screening
• Be able to understand and provide written informed consent
• Potentially be age- and/or sex-matched with a transplant patient
• Subjects in Cohort HL must additionally be willing and able to return to the NIH Dental Clinic for scheduled visits across 1 year and participate in evaluations including saliva collection, oral swab collection, and completion of PRO forms

You may not qualify if:

• Subjects in any Cohort will be excluded if they are pregnant, as pregnancy can have a confounding effect on the oral microbiome
• Subjects in Cohort NT may not participate in the study if, in the opinion of the NIH transplant team, participation in the protocol would not be safe or in the subject s best interest
• Subjects in Cohorts HL and Cohort HS may not participate in the study if they meet either of the following criteria:
• Have any acute oral mucosal or dental infection requiring multiple dental appointments or specialist referral for treatment; uncontrolled chronic disease; active systemic illness; are currently undergoing orthodontic treatment; history of frequent opportunistic infection; autoimmune disease; immunosuppressive medications; or prior organ, bone marrow, or peripheral blood stem cell transplant
• Are taking medications or are on other ongoing therapies that have a known or suspected major impact on the oral microbiome
• Subjects in Cohort HL additionally may not participate if they are planning to become pregnant within the next year or refuse to have oral biopsies.

Sponsors & Collaborators

• National Institute of Dental and Craniofacial Research (NIDCR): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Publications (4)

• Mays JW, Fassil H, Edwards DA, Pavletic SZ, Bassim CW. Oral chronic graft-versus-host disease: current pathogenesis, therapy, and research. Oral Dis. 2013 May;19(4):327-46. doi: 10.1111/odi.12028. Epub 2012 Oct 28.

  PMID: 23107104 BACKGROUND

• Cooke KR, Luznik L, Sarantopoulos S, Hakim FT, Jagasia M, Fowler DH, van den Brink MRM, Hansen JA, Parkman R, Miklos DB, Martin PJ, Paczesny S, Vogelsang G, Pavletic S, Ritz J, Schultz KR, Blazar BR. The Biology of Chronic Graft-versus-Host Disease: A Task Force Report from the National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease. Biol Blood Marrow Transplant. 2017 Feb;23(2):211-234. doi: 10.1016/j.bbmt.2016.09.023. Epub 2016 Oct 3.

  PMID: 27713092 BACKGROUND

• Jagasia MH, Greinix HT, Arora M, Williams KM, Wolff D, Cowen EW, Palmer J, Weisdorf D, Treister NS, Cheng GS, Kerr H, Stratton P, Duarte RF, McDonald GB, Inamoto Y, Vigorito A, Arai S, Datiles MB, Jacobsohn D, Heller T, Kitko CL, Mitchell SA, Martin PJ, Shulman H, Wu RS, Cutler CS, Vogelsang GB, Lee SJ, Pavletic SZ, Flowers ME. National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: I. The 2014 Diagnosis and Staging Working Group report. Biol Blood Marrow Transplant. 2015 Mar;21(3):389-401.e1. doi: 10.1016/j.bbmt.2014.12.001. Epub 2014 Dec 18.

  PMID: 25529383 BACKGROUND

• Leinbach LI, Boroumand S, Masuch LR, Nguyen JT, Ganesan SM, Mays JW. Oral Health Characteristics 2 Years following Allogeneic Cell Transplant. J Dent Res. 2025 Sep;104(10):1061-1068. doi: 10.1177/00220345251329351. Epub 2025 May 12.

  PMID: 40353516 DERIVED

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Graft vs Host Disease; Autoimmune Diseases; Ulcer

Condition Hierarchy (Ancestors)

Immune System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Jacqueline W Mays, D.D.S.

  National Institute of Dental and Craniofacial Research (NIDCR)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Licia R Masuch

CONTACT

(301) 335-0113; licia.masuch@nih.gov

Jacqueline W Mays, D.D.S.

CONTACT

(301) 496-8800; jacqueline.mays@nih.gov

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 26, 2018
• First Posted: July 27, 2018
• Study Start: November 15, 2018
• Primary Completion (Estimated): March 3, 2037
• Study Completion (Estimated): March 3, 2037
• Last Updated: April 14, 2026

Record last verified: 2026-01-30

Locations

US (1)