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Table · dataset · 2026

Table 1_Regression of severe sclerotic chronic graft-versus-host disease after a second haploidentical hematopoietic stem cell transplantation: a case report.docx

Listed in figshare and Loughborough Research Repository — shown once because both records carry DOI 10.3389/fmed.2026.1929283.s001

<p>Sclerotic chronic graft-versus-host disease (Scl-cGVHD) is a disabling fibrotic manifestation of cGVHD and frequently exhibits poor responsiveness to conventional immunosuppressive therapy.

Description

Therapeutic options remained limited, particularly in pediatric patients. An 8-year-old boy with relapsed acute myeloid leukemia underwent matched sibling donor allogeneic hematopoietic stem cell transplantation (allo-HSCT) in January 2021.

Four months later, he developed progressive cutaneous cGVHD that evolved into severe Scl-cGVHD, with diffuse skin thickening, painful fissures, ulceration, and multiple joint contractures, loss of independent ambulation, and complete dependence in activities of daily living. Skin biopsy demonstrated extensive dermal fibrosis, collagen deposition, and increased α-smooth muscle actin (α-SMA) expression. Despite multiple lines of immunosuppressive and targeted therapy, including corticosteroids, calcineurin inhibitors, mycophenolate mofetil, ruxolitinib, rituximab, and other agents, the disease progressed, with the modified Rodnan skin score (mRSS) reaching 35.

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Because of refractory disease and disabling disease, a second haploidentical HSCT from his father was performed in September 2022. Following transplantation, disease progression ceased and gradual clinical improvement occurred. At 12 months, the mRSS decreased from 35 to 2, with substantial recovery of skin flexibility and joint mobility.

Repeat skin biopsy showed reduced collagen compactness and markedly decreased interstitial α-SMA expression, consistent with attenuation and remodeling of dermal fibrosis. Serial immune monitoring revealed progressive recovery of B- and NK-cell compartments and sustained T-cell reconstitution after the second transplantation, in contrast to prolonged B-cell depletion and limited NK-cell recovery after the first transplant.

At 24 months, complete donor chimerism persisted, leukemia remained in remission, the mRSS reached 0, and the patient regained independent walking and activities of daily living. This case describes sustained clinical regression and histopathological attenuation of severe, established Scl-cGVHD following a second haploidentical HSCT. The temporal association with immune reconstitution raises the possibility that restoration of immune homeostasis may have contributed to disease remodeling.

However, causality cannot be established from a single case. Given the substantial risks associated with second allo-HSCT, this approach should not be considered standard therapy and may be considered only in exceptional, highly selected patients after multidisciplinary risk-benefit assessment.</p>

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Disease 75% · Tabular 65%
Provenance · 2 source records, 32 field assertions
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