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Abstract
Background
Etanercept is a recombinant human soluble tumor necrosis factor-alpha (TNF-α) receptor fusion protein that inhibits TNF-α, a major mediator in the pathogenesis of graft-versus-host disease (GVHD). The purpose of our study was to evaluate the safety and efficacy of etanercept therapy in children with steroid-refractory acute GVHD (aGVHD) (n=5) and chronic GVHD (cGVHD) (n=3).
Methods
Five males and 3 females were enrolled and their median age was 14.4 years (range, 2.1∼18.8). Etanercept 0.4 mg/kg per dose (maximum dose, 25 mg) was given subcutaneously twice weekly for 4 weeks followed by 0.4 mg/kg per dose (maximum dose, 25 mg) weekly for 4 weeks. At the time of initiation of etanercept, 5 patients had aGVHD grade III to IV (III=4, IV=1) and 3 patients had moderate to severe cGVHD (moderate=1, severe=2).
Results
Overall, 6 of 8 patients (75%) responded to the treatment with etanercept, including 5 patients with aGVHD [n=3 complete response (CR), n=2 partial response (PR)] and 1 patient with cGVHD [n=1 PR, n=2 no response (NR)]. Clinical responses were most commonly seen in patients with refractory gut aGVHD. CMV reactivation occurred in 2 patients, bacterial infection in 1 patient, and fungal infection in 1 patient.
References
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Table 1.
Characteristics of the patients and donors
Abbreviations: P, patient; D, donor; M, male; F, female; HLA, human leukocyte antigen; GVHD, graft-versus-host disease; AML, acute myeloid leukemia; SAA, severe aplastic anemia; WAS, Wiskott-Aldrich syndrome; DLBL, diffuse large B cell lymphoma; CML, chronic myeloid leukemia; U, unrelated; R, related; BM, bone marrow; CB, cord blood; PB, pheripheral blood; Bu, busulfan; Flu, fludarabine; Cy, cyclophosphamide; ATG, anti-thymocyte globulin g; CsA, cyclosporine A; MTX, methotrexate; PD, prednisolone; MMF, mycophenolate mofetil.
Table 2.
Days of immunosuppressive treatments
Table 3.
Treatment with etanercept in patients with aGVHD
Table 4.
Treatment with etanercept in patients with cGVHD
Table 5.
Clinical outcomes of the patients treated with etanercept
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