TANDEM Meeting 2026

The Congress Connection
Congress Overview

Belumosudil demonstrated efficacy in severe sclerotic skin and fascial cGVHD, with higher response rates observed in patients with NIH score 2 vs 3 (including >25% complete response for skin score 2) across two prospective studies.¹

• The study assessed the efficacy of belumosudil in sclerotic cGVHD patients, along with response rates using 2014 NIH criteria and examined how patient vs. clinician perceptions impact treatment outcomes, corticosteroid use, and quality of life.
• Baseline Characteristics: Of 159 patients with sclerotic cGVHD (73.6% severe), 133 (83.6%) had sclerotic skin involvement (33 with score 2, 100 with score 3) while 26 (16.4%) had only joints/fascia involvement.
• Primary endpoint (skin): Overall skin response rate was 28.3% (CR 10.7%; PR 17.6%).
• Response rates cross NIH skin score categories were 32.4% (score 2) and 28.3% (score 3), with overall median time to response 12.0 weeks (range 3.7–146.4).
• Joints/fascia-specific overall response rate was 62.3%, with 56.4% (score 1), 72.7% (score 2), and 70.6% (score 3); along with median time to response was 8.1 weeks in all groups.

Belumosudil in combination with ECP demonstrated meaningful clinical outcomes and favourable safety in patients with cGvHD in the ROCKstar trial.²

• This post hoc analysis of the ROCKstar trial compared outcomes in patients receiving belumosudil (200 mg once or twice daily) with or without concomitant ECP, assessing ORR, TTR, DOR, and LSS scores using 2014 NIH consensus criteria, with descriptive summary of TEAEs.
• ORR was 81.8% in the belumosudil + ECP group vs 72.2% in the belumosudil-alone group.
• A ≥7-point reduction in LSS score observed in 68.2% of patients receiving belumosudil + ECP vs 58.3% without ECP; ≥7-point reductions on two consecutive occasions were 43.2% vs 44.4%, respectively.
• TEAEs were higher in the belumosudil + ECP group than in the belumosudil-alone group, particularly for infections/infestations (79.5% vs 57.4%) and metabolism/nutrition disorders (56.8% vs 47.2%). Several other events—including fatigue, diarrhoea, cough, headache, hypertension, and arthralgiaoccurred at numerically lower rates in patients receiving ECP.

The generative AI based approach leveraging large patient electronic-health-record identified a high prevalence and progressive pattern of fibrosis in cGVHD, by detecting fibrosis manifestations that highlighted clinical value of early, AI enabled detection to support timely intervention and potentially minimize irreversible fibrotic damage.³

• At baseline, 142 patients out of 925 (15%) had fibrosis-specific manifestations which increased to 432 (47%) during follow-ups; 32% ↑ from baseline.
• Progression from fibrosis suggestive to fibrosis specific terms occurred during follow up.
• Of 503 patients had fibrosis suggestive terms at baseline; 53% progressed to fibrosis specific findings, demonstrating substantial disease evolution identified through AI enabled EHR analysis.
• Highest organ specific conversion rate was observed in skin (37%) followed by lung (25%), joint/fascia (22%), and GI tract (18%) that showed multisystem evolution of fibrosis in cGVHD.

This qualitative interview study showed that adults with cGVHD experience persistent multiorgan symptoms and treatment-burdens related to administration, side effects, cost or insurance coverage, adherence underscoring the need for investigation on convenience, time to onset, effectiveness, and steroid requirements as factors influencing treatment choice.⁴

• 15 patients with cGvHD ≥3 years and aged ≥18 years were interviewed. Participants reported multisystem (≥3 organs) cGVHD symptom with treatments since diagnosis such as, oral, topical, IV, injection, SC, eye drops, mouth rinse or toothpaste, and ECP, commonly with eyes 93% and skin-hair-nails 87% followed by energy level, respiratory, muscle or joint, GI, genitals or sex organs and liver-related.
• Alongside, high treatment burden included frequency (n=6), cost/coverage issues (n=5), travel time (n=3), and multiple hospital visits (n=2), difficulty in treatment adherence (n=4).
• Findings addressed evidence gaps in understanding patient satisfaction with treatment preferences promoting additional investigation into clinical and non clinical aspects to support patient care

This Belgian real-world single centre retrospective study evaluated the practices and presentation of GVHD prophylaxis after first allogenic HSCT (n=259) and showed that moderate/severe cGVHD continues to be a debilitating condition with significant impact on OS, particularly with lung involvement.⁵

• Calcineurin inhibitor + MMF was the predominant prophylaxis regimen (81.9%, n=212), followed by MTX (13.9%, n=36) and other approaches.
• At median of 18.8 (IQR, 7.9–38.2) months follow-up, HR for OS by
• Presence of cGVHD was 1.14 (95% CI: 0.72–1.81, P=0.56)
• Moderate/severe cGVHD was 1.79 (95% CI:1.14–2.80, P=0.01)
• Involvement of lung was 2.59 (95% CI: 1.21–5.53, P=0.01)
• Incidence of symptoms at onset and maximal severity were most frequent in skin, mouth, eyes, GI tract, liver followed by lungs, genitals, and muscle/facia.

Management of GvHD is shifting toward gut focused repair in aGvHD, diversified targeted agents in cGvHD, and smarter prophylaxis (including PTCy dose optimization and novel graft/sculpting or ruxolitinib based regimens).

• aGvHD advances: Ruxolitinib remains standard but has <40% day-56 responses and no mortality benefit; 6 GLP-2 analogs (apraglutide) combined with ruxolitinib showed 54.8% ORR with sustained responses through day 90; 7 microbiome approaches (phase 3 FMT) show promising response durability and organ spillover effects; 8 adult MSC trials (CTN 2303) are opening.
• cGvHD innovations: Four FDA approved options (ibrutinib, belumosudil, ruxolitinib, axatilimab) with next gen ROCK2 and dual JAK/ROCK inhibitors (e.g., rovidacitinib, GV 101/TDI 01) and CSF1R inhibitors (vimseltinib) improving response rates and enabling steroid sparing; biomarker guided drug selection is a key future need.
• Prophylaxis optimization: Beyond PTCy, strategies include TAC/methotrexate+ruxolitinib (BMT CTN 2203), PTCy dose de escalation (25 mg/kg), PTCy+ruxolitinib or abatacept (NMDP Accelerate), and ORCA T graŌ sculpting showing superior GRFS versus TAC/methotrexate.

Chronic GVHD management has advanced significantly beyond steroids, with four FDA-approved therapies now enabling faster, more durable, and more organ-specific responses especially for patients with fibrotic disease.

• Growing therapeutic arsenal: Ibrutinib (BTK inhibitor)9 , ruxolitinib (JAK1/2)6 , belumosudil (ROCK2)10 , and axatilimab (CSF-1R) 11 mainly target inflammatory and fibrotic pathways, showing ORRs of 67%, 50% vs 25% BAT, 76%, and 74%, respectively leading to improved responses compared with steroids alone.
• Real-world treatment strategy: Ruxolitinib provides rapid inflammatory control; belumosudil helps across multiple organs; axatilimab shows strong efficacy in heavily pretreated and fibrotic disease.
• Personalized-therapeutic approach: Clinicians must tailor therapy based on toxicity profiles, comorbidities, organ response patterns, logistics (oral vs IV), and quality-of-life goals to move patients toward steroid-free, durable remission.

Pulmonary cGvHD, primarily BOS, remains a severe post transplant complication requiring early detection and structured PFT monitoring. Steroids plus FAM remain first line therapy, while ibrutinib, ruxolitinib, belumosudil, and axatilimab show clinical meaningful lung specific activity, especially in mild-moderate disease. Advanced cases may require combination therapy, ECP, or lung transplantation, emphasizing the need for BOS focused prospective studies.

• Ruxolitinib, belumosudil, and axatilimab demonstrate the strongest lung-specific efficacy among available therapies, with axatilimab achieving the highest reported pulmonary response rates, including in severe BOS, while ruxolitinib and belumosudil show meaningful benefits primarily in mild-moderate disease.
• First-line management with systemic corticosteroids plus the FAM regimen remains the standard of care, offering early stabilization of lung function and supporting steroid tapering, though long-term azithromycin use requires caution due to malignancy risk.
• Progressive or refractory pulmonary cGvHD may require combination therapies, adjunctive modalities such as ECP, or lung transplantation, underscoring the critical need for BOS-specific, prospective clinical trials and biomarker-driven treatment optimization.