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Tandem Meetings Transplantation and Cellular Therapy Meetings of ASTCT® and CIBMTR®

The Congress Connection: cGvHD and Transplant Key Takeaways in 2025

Development and psychometric validation of a new pediatric chronic GvHD (cGVHD) symptom scale for children and caregiver-proxies
Williams K (USA)

Patient-reported outcomes are important and capture the patient perspectives directly. Although there is a cGvHD symptom scale for adults (the Lee symptom scale), there is no scale for children with cGvHD. The study aims to refine and validate a PCSS based on the domains from Lee symptom scale.1

  • Project 1 – concept elicitation interviews to define symptom selection, terminology/phrasing, ease of response, clarity, relevance, and response choice (N=60, 40 completed; childcaregiver dyads).
  • Preliminary results: Asking children to rephrase items led to item pruning and refinement e.g., ‘my eyes stinging or burning’, ‘using an oxygen mask to help me breathe’, ‘not feeling hungry’. Child and caregiver debriefing interviews ensured comprehension and clarity of items in each age category. While the 5-7 years old generally understood the symptoms, they felt difficulty in comprehending subtle distinctions for e.g., skin hardening versus skin thickening.
  • Project 2 is planned to evaluate psychometric testing of scale validity, reliability, and responsiveness (N=120; child-caregiver dyads). Also, determine the lowest age for Lee-scale comprehension by adolescents.
  • Current status - recruiting patients.

Chronic GvHD: Nailing the diagnosis & exam
Epling P (USA)

cGvHD is a leading cause of NRM in post-bone marrow transplant patients with a high mortality rate of 37.8%2 presenting with early skin changes, dry, itchy eyes, and oral symptoms, and requires careful and accurate diagnosis using NIH scoring system and diagnostic imaging to manage its complex interactions and complications.

  • Early diagnosis and symptoms: cGvHD can present with early skin changes, dry, itchy eyes (occurring in 40–60% of allogeneic transplant patients), and oral symptoms like inflammation and ulcerations, which can lead to severe mucosal pain and decreased nutritional status.
  • NIH scoring data3: The NIH scoring system for cGvHD includes performance scales (e.g., Karnofsky score), skin involvement (e.g., maculopapular rash, sclerotic features), and organspecific symptoms (e.g., dry eyes, GI symptoms). For example, a performance scale of 80 gives 1 point, skin involvement of 50% BSA gives 2 points, and mild dry eyes not requiring frequent eye drops gives no points.
  • Key diagnostic Imaging and physical examination: Diagnostic imaging for GI cGvHD can reveal multifocal small bowel strictures, fluid and gas collections indicating complications like cecal perforation, and mucosal changes in the colon.4 Musculoskeletal symptoms include joint tightness and muscle weakness, often requiring a combination of physical therapy and medical interventions.5

Single center experience of belumosudil in children <12 years, adolescents and young adults
Ibrahimova A (USA)

Belumosudil was well-tolerated in children and young adults with cGvHD, though the ORR was lower than published data, likely due to the severity of cGvHD in the study population.

  • This retrospective study reviewed charts of 16 patients (<12y: 2; 12–18y: 9; >19y: 5) treated with belumosudil for cGvHD from August 2021 to July 2024. Severe cGvHD was reported in 14 patients. The median treatment duration was 177 days.
  • The ORR was 44%, with PR in 7 patients NR in 9. Among the 2 children <12 years, 1 had a PR and 1 had NR. Organ-specific PRs were observed in skin, mouth, eyes, lungs, genitourinary, joints/fascia, GI tract, and liver. Corticosteroids were successfully weaned in 9 patients. Seven patients discontinued belumosudil due to lack of response, and 1 due to concurrent AEs.
  • AEs: Bacterial infections, hepatotoxicity, thrombocytopenia, and QTc prolongation.

Real world outcomes of belumosudil for treatment of chronic graft-versus-host disease: A single center experience
Modi B (USA)

A single center study showed that belumosudil is safe and effective for use in real world settings after patients have progressed through multiple lines of therapy, including ruxolitinib. Combination with ruxolitinib significantly increased the risk of infections without improving the ORR. Further research is necessary to identify predictors of response to belumosudil.

  • A retrospective analysis assessed the safety and efficacy of belumosudil in 45 patients (median age 59 years, range 19-78) who received belumosudil at FDA-approved doses from August 2021 to November 2022.
  • Multiorgan involvement: Patients with cGVHD showed multiorgan involvement affecting skin (75.6%), eyes (48.9%), mouth (46.7%), and joints (37.8%). Median follow-up at the time of analysis for all patients was 22.9 months.
  • ORR: The best ORR observed was 46.7% (complete response: 6.7% + partial response: 40%) with a median time to best response of 108.5 days. ORR was 31.1% at 6 months and 33.4% at 12 months. Steroid dose reduction and infection rates: About 29% of the patients reduced their corticosteroid dose, and 20% of patients discontinued steroids. Patients on combination therapy with ruxolitinib and belumosudil had higher infection rates (61%) compared to those on belumosudil monotherapy (23%; p=0.01).
  • Steroid dose reduction and infection rates: About 29% of the patients reduced their corticosteroid dose, and 20% of patients discontinued steroids. Patients on combination therapy with ruxolitinib and belumosudil had higher infection rates (61%) compared to those on belumosudil monotherapy (23%; p=0.01).

Real-world patient characteristics and treatment patterns in patients with chronic graftversus- host disease receiving belumosudil in the United States
Yu J (USA)

Real-world cGvHD treatment patterns, including belumosudil, are underexplored, emphasizing the need to study patient characteristics and treatment sequencing.

  • This retrospective study analyzed 181 cGvHD patients who initiated belumosudil (2019–2023) using the Komodo Healthcare Map. Patients required ≥6 months of health insurance coverage pre- and post-initiation (unless deceased). Treatment duration, lines of therapy (LOTs), and prior/concurrent treatments were assessed.
  • The mean (SD) age was 48.2 (17.4) years, with 56.9% male. Patients started belumosudil a median of 489 days post-cGvHD diagnosis, mainly in LOT 3 or later (81.8%). Prior treatments included ruxolitinib monotherapy (22.5%) and CNI. Belumosudil was used as monotherapy/corticosteroids (45.9%) or combination therapy (54.1%). The median duration of belumosudil LOT was 133 days (IQR; 60–276). By follow-up, 39.8% had discontinued, 27.1% switched, and 20.4% added therapies.
  • Belumosudil was largely used after ≥3 prior LOTs, likely due to approval timing. Further research is needed to evaluate treatment sequencing and long-term outcomes.

Patient experiences with chronic graft-versus-host disease and its treatment in the United States: a retrospective social media listening study
Cowden MA (USA)

SML reveals significant physical and mental impacts of cGvHD and highlights the need for improved public awareness and treatment access.

  • Overall, 8.4% (n=107) of social media posts describing compounded physical and emotional burdens experienced by patients with cGvHD. The physical symptoms often exacerbate the emotional toll, creating a challenging cycle for patients. Effective management of these symptoms is crucial for improving patient’s QoL.
  • Positive relationships with HCPs are crucial in managing cGvHD. 10.9% (n=139) of posts highlighted the importance of HCP expertise and compassion, suggesting that effective communication and personalized treatment could help mitigate the disease's impact.
  • Treatment regimens for cGvHD are complex and expensive. 10.3% (n=132) of posts mentioned difficulties in accessing new treatments, while 8.3% (n=106) noted the financial burdens associated with care. These challenges underscore the need for better treatment options and support systems.

Harnessing machine learning to predict chronic GvHD: A novel risk stratification score
Ramgopal A (Panama)

cGvHD is a major complication of HSCT, and early prediction can improve management.

  • This study used ML to develop a predictive model for cGvHD risk stratification.
  • De-identified patient data from CIBMTR (2008–2017) was used to train ML models, optimized through 50 trials with five-fold cross-validation. Accuracy metrics and a confusion matrix assessed performance, while feature importance analysis identified key predictors.
  • The ML model achieved 73.2% sensitivity but had lower specificity (44.25%), leading to misclassification of some non-cGvHD cases. The Random Forest model identified anti-thymocyte globulin (17.5%) and aGvHD (12%) as top predictors, contributing nearly 30% to model performance.
  • With an overall accuracy of 59%, the model shows moderate predictive capability. Enhancing specificity and expanding data could improve accuracy, supporting early risk stratification and optimized HSCT treatment.

Acute and chronic graft versus host disease (GvHD) management in the Eastern Mediterranean region: Result of the Worldwide Network for Blood & Marrow Transplantation (WBMT) global GvHD survey
Muhsen IN (USA)

Refractory GvHD remains a major challenge in the EM region. While treatment generally follows guidelines, survey findings stress the importance of increased trial access, multidisciplinary support and better management strategies.

  • This survey assessed aGvHD and cGvHD management across 30 programs in 26 institutions from 11 EM countries (December 2022–June 2023).
  • aGvHD: Initial prednisone doses were 1 mg/kg/day (grade IIa) and 2 mg/kg/day (grade IIb). Ruxolitinib (83%) was the most common second-line therapy, followed by CNI (47%) and high-dose steroids (43%). Only 7% of programs included steroid-refractory aGvHD patients in trials, while 30% lacked SOP.
  • cGvHD: Systemic steroids were the first-line treatment. For steroid-refractory cases, ruxolitinib (80%) was most used, followed by CNI (67%) and mycophenolate mofetil (53%). Only 20% had multidisciplinary teams, 10% enrolled steroid-refractory patients in trials, and 43% lacked an SOP.

Association of patient-reported outcomes with survival and graft versus host disease in allogeneic hematopoietic cell transplant recipients
Ardila V (USA)

Pre-HCT depression, distress, and QoL are significant predictors of post-HCT outcomes, highlighting the importance of routine PRO assessments in clinical practice.

  • This retrospective study examined the association of pretransplant PROs with survival and GvHD in 583 patients who underwent their first allogeneic HCT from 2015 to 2022. PROs were measured using the PHQ-9, NCCN DT, and PROMIS PH and MH.
  • Median (IQR) pre-HCT PHQ-9 scores were 3 [1–6], with 20.5% reporting moderate to severe depression; median NCCN DT scores were 1.0 [0–3.0], with 24.2% reporting moderate to severe distress. Median PROMIS PH and MH T-scores were 47.7 [39.8–54.1] and [43.5–53.3], respectively.
  • Higher pre-HCT depression and distress were significantly associated with increased aGvHD and trends towards higher incidence cGvHD, NRM, and lower OS.

Impact of graft-versus host disease following allogeneic hematopoietic cell transplantation on leukemia free survival in hematologic malignancies: A CIBMTR Analysis
Qayed M (USA)

This study found that neither aGVHD nor cGVHD significantly impacted relapse rates in children with AML or ALL post-HCT, regardless of disease risk. However, severe aGVHD was associated with inferior LFS and higher HR for LFS.

  • In this study, 2,328 pediatric patients who received their first allo-HCT for ALL or AML following myeloablative conditioning between 2008–2017 were included.
  • At day 100, 47% of patients with AML developed aGvHD (grades 1-4: 17%, 19%, 8%, 3%) and 27% had cGvHD (limited 10%, extensive 17%), while 52% of patients with ALL developed aGvHD (grades 1-4: 17%, 21%, 9%, 4%) and 30% had cGvHD (limited 11%, extensive 19%).
  • The cumulative incidence of relapse for AML was around 20%, with no significant differences in relapse rates based on GvHD grade. In AML patients with low/intermediate or high/very high DRI, no significant differences in cumulative incidence of relapse were found by GvHD severity. In patients with high or very high DRI also, no difference in cumulative incidence of relapse by severity of GvHD was found. However, severe aGvHD was associated with inferior LFS and higher HR for LFS.

Abbreviations
AE, adverse event; aGvHD, acute graft-versus-host-disease; ALL, acute lymphoblastic leukemia; allo-HCT, allogenic hematopoietic cell transplantation; AML, acute myeloid leukemia; BSA, bovin serum albumin; CIBMTR, Center for International Blood and Marrow Transplant Research; cGvHD, chronic graft-versus-host-disease; CNI, calcineurin inhibitors; DRI, disease relapse index; EM, Eastern Mediterranean; FDA; Food and Drug Administration; GI, gastrointestinal; GvHD; graft-versus-host disease; HCP, healthcare professional; HCT, hematopoietic cell transplant; HR, hazard ratio; HSCT, hematopoietic stem cell transplantation; IQR, interquartile range; LFS, leukemia-free survival; MH, mental health; ML, machine learning; NCCN DT, National Comprehensive Cancer Network Distress Thermometer; NIH, National Institute of Health; NR, no response; NRM, nonrelapse mortality; ORR, overall response rate; OS, overall survival; PCSS, pediatric cGvHD symptom scale; PH, physical health; PHQ-9, Patient Health Questionarie-9; PR, partial response; PRO, patient-reported outcome; PROMIS, Patient-Reported Outcomes Measurement Information System; PTSD, post traumatic stress disorder; QoL, quality of life; Qtc, corrected QT interval; SML, social media listening; SOP, standard operating procedure.

  1. Mitchell SA et al. Front. Psychol. 2024:14:1243005; 1-13.;
  2. DeFilipp Z, et al. Blood Adv. 2021;5(20):4278-4284.;
  3. Jagasia MH, et al. Biol Blood Marrow Transplant. 2015;21(3):389-401.e1.;
  4. Lubner MG, et al. Radiographics. 2017;37(5):1483-1503.;
  5. Carpenter PA. Blood. 2011;118(10):2679-2687.
MAT-SA-2500220-v1.0-03/2025