Ruxolitinib in steroid refractory graft-vs.-host disease: a case reportReportar como inadecuado

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Journal of Hematology and Oncology

, 9:67

First Online: 08 August 2016Received: 07 June 2016Accepted: 03 August 2016DOI: 10.1186-s13045-016-0298-6

Cite this article as: Maffini, E., Giaccone, L., Festuccia, M. et al. J Hematol Oncol 2016 9: 67. doi:10.1186-s13045-016-0298-6


BackgroundAllogeneic hematopoietic stem cell transplantation HSCT is potentially curative in a variety of hematological malignancies. Graft-vs.-host disease GvHD remains a life-threatening complication. Standard treatment is high-dose HD corticosteroids. Steroid-refractory SR GvHD is associated with poor prognosis. At present, second-line treatment is ill-defined and includes a number of agents. Novel insights into the pathophysiology of acute GvHD aGvHD highlight the relevant role of the host inflammatory response governed by several kinase families, including Janus kinases JAK1-2. Ruxolitinib, a JAK1-2 inhibitor approved for intermediate-2-high-risk myelofibrosis, was recently employed in SR-GvHD with encouraging overall response rates. Clinical experience however remains limited.

Case presentationA 51-year-old male with refractory anemia with excess blast type-2 underwent a myeloablative allogeneic HSCT from a 9-10 HLA-matched unrelated donor after conditioning with busulfan and cyclophosphamide. GvHD prophylaxis consisted of cyclosporine, methotrexate, and thymoglobulin. CD34 cells-kg infused were 8.69 × 10 kg. On day 29, the patient developed overall grade IV aGvHD with biopsy proven stage IV gastrointestinal GI GvHD refractory to HD corticosteroids. Patient conditions rapidly deteriorated and became critical despite the addition of mycophenolate mofetil and budesonide. On day 33, Ruxolitinib was started, and on day 39 the patient clinical conditions gradually improved. Complete resolution of aGvHD was also confirmed by histology on day 54.

ConclusionsAt 5 months from HSCT, the patient is well and in continuous hematological complete remission without flare of GvHD. Ruxolitinib was discontinued on day 156. Ruxolitinib is feasible and effective in SR-aGvHD though large prospective clinical trials are warranted.

KeywordsAllogeneic hematopoietic stem cell transplant HSCT Steroid-refractory graft-vs.-host disease SR-GvHD Ruxolitinib Regulatory T cells Treg Proinflammatory cytokines Case report  Download fulltext PDF

Autor: Enrico Maffini - Luisa Giaccone - Moreno Festuccia - Lucia Brunello - Ilaria Buondonno - Dario Ferrero - Mario Boccadoro -


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