Nilotinib as Salvage Therapy In Patients with Chronic Graft Versus Host Disease

Abstract 2329

The mainstay of treatment for Chronic Graft Versus Host Disease (cGVHD) is steroids, but there are limited treatment options for steroid refractory cGVHD particularly in its sclerotic and fibrotic form (ScGVHD). We initially reported the efficacy and safety of imatinib mesylate (IM), a first generation tyrosine kinas' inhibitor, as a salvage therapy for ScGVHD patients (Magro L, et al. Blood, 2009). However, about 50% of the patients (Pts) became refractory or intolerant to IM. Nilotinib, a second generation tyrosine kinas' inhibitor, is labelled for IM resistant and advanced Chronic Myeloid leukemia and could be an attractive alternative to IM especially in refractory and intolerant patients.

We studied the clinical outcomes of 7 Pts who developed extensive ScGVHD at our institution. Among them, 5 had received prior IM. Patients' characteristics and transplantation modalities are summarised in table 1. Acute and cGVHD was scored according to standard criteria. All pts but one failed at least 3 lines of prior systemic immunosuppressive therapy.

CGVHD features, responses to nilotinib and patients' outcomes are described in table 2.

To our knowledge, this is the first report relating the impact of nilotinib on cGVHD setting. Although, tolerance to nilotinib has not been as good as we could expect, this drug appeared to be effective with manageable side-effects in some patients. One of the limitations of this study, is that nilotinib was used in patients with advanced cGVHD. Prospective studies are, therefore, warranted to investigate the efficacy and the tolerance of nilotinib in patients with less advanced disease.