{
  "abstract": "Background Chronic graft-versus-host disease (GvHD) is a common long-term complication following allogeneic haemopoietic progenitor cell transplantation, with the incidence dependent on donor type, stem cell source, conditioning regimen and patient population. 1 Oral GvHD can affect up to ~80% of patients, even with prophylactic immunosuppressive therapy and can severely diminish quality of life by causing pain, limiting oral intake and increasing the risk of malnutrition and secondary malignancies.2 3 Given these risks, topical treatments are recommended to be initiated at any stage of oral involvement.A 5-year-old patient underwent 7/8, 8/10 mis-matched umbilical cord blood transplant, with ciclosporin and mycophenolate mofetil as GvHD prophylaxis. Post-transplant, he developed grade 3 skin GvHD, significant ocular involvement and marked oral mucosal inflammation. Systemic corticosteroids were initiated in the setting of worsening skin GvHD, with ongoing mouthcare. The patient was also started on extracorporeal photopheresis (ECP) and ruxolitinib was later commenced as a steroid-sparing agent as part of the management.Pharmacy Contributions There are currently no licensed intra-oral treatments for oral GvHD, necessitating the off-label and off-license use of topical agents. In collaboration with oral medicine specialists at University College London Hospital (UCLH) Eastman Dental Hospital, pharmacists played a central role in evaluating treatment options, including beclomethasone 50micrograms/actuation inhaler, pimecrolimus 1% cream and clobetasol propionate 0.05% cream for use in the oral cavity – topical application to the affected areas of gums and cheeks using a gauze and leaving for 5-10 minutes to allow exposure of product. A comprehensive literature review was conducted to determine appropriate dosing regimens and assess the safety and efficacy of these agents. Pharmacists ensured adherence to local clinical governance procedures and provided detailed counselling to the patient’s family. This included discussions on the unlicensed and off-label nature of the treatments, potential side effects, administration techniques, and the intended therapeutic outcomes. Pharmacists also supported ongoing monitoring for tolerability and adverse effects throughout the treatment course.Outcome The patient responded to topical pimecrolimus but developed gum ulceration. This was then switched to clobetasol propionate cream, with clinical improvement allowing for the tapering of clobetasol propionate over 4-weeks. Pimecrolimus was restarted later. No adverse reactions were observed. Pimecrolimus, which has demonstrated safety and efficacy in the treatment of oral lichen planus, 4 proved to be a valuable option in this case. However, due to age and palatability, adherence to topical therapy was a challenge, requiring additional support from the multidisciplinary team (MDT).Lessons Learned Pharmacists play a pivotal role in the safe and effective management of oral GvHD, particularly when treatment involves unlicensed and off-label medicines. Pimecrolimus, supported by emerging evidence and clinical experience appears to be a safe and effective topical option when used alone or in combination with other agents. Successful outcomes rely on MDT collaboration, robust clinical governance and clear communication with patients and families. Pharmacy expertise in pharmacokinetics, risk assessment and patient/family counselling is essential in navigating complex therapeutic decisions and optimising care.References David D and Sroussi H. Oral chronic graft-versus-host disease. Frontiers in Oral Health 2022;3:903154.Nguyen JT, Jessri M, Costa-da-Silva AC, et al. Oral chronic graft-versus-host disease: pathogenesis, diagnosis, current treatment, and emerging therapies. International Journal of Molecular Sciences 2024;25:10411.Treister N, Duncan C, Culter C, et al. How we treat oral chronic graft-versus-host disease. Blood 2012;120:3407–3418.Passeron T, Lacour J-P, Fontas E, et al. Treatment of oral erosive lichen planus with 1% pimecrolimus cream: a double-blind, randomised, prospective trial with measurement of pimecrolimus levels in the blood. Archives of Dermatology 2007;143(4):472–476.",
  "authors": [
    {
      "affiliations": [
        "Great Ormond Street Hospital for Children, UK"
      ],
      "name": "Ka Yu Yung"
    },
    {
      "affiliations": [
        "Great Ormond Street Hospital for Children, UK"
      ],
      "name": "Rebecca O’Neill"
    }
  ],
  "title": "P42 Management of oral graft-versus-host disease in a paediatric patient",
  "uid": "49939e94-07b4-50f5-846a-89ff0892ebd6"
}