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      Response to ruxolitinib in patients with intermediate-1-, intermediate-2-, and high-risk myelofibrosis: results of the UK ROBUST Trial.

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          Myelofibrosis is characterized by splenomegaly and debilitating constitutional symptoms that negatively impact patients' quality of life. ROBUST, a UK, open-label, phase II study, evaluated the safety and efficacy of ruxolitinib in patients with myelofibrosis (N = 48), including intermediate-1 risk patients. The primary composite endpoint was the proportion of patients achieving treatment success [≥ 50% reduction in palpable spleen length and/or a ≥ 50% decrease in Myelofibrosis Symptom Assessment Form Total Symptom Score (MF-SAF TSS)] at 48 weeks. This was the first time that efficacy of ruxolitinib in myelofibrosis has been evaluated based on these criteria and the first time the MF-SAF was used in a population of patients solely from the United Kingdom. Overall, 50% of patients and 57% of intermediate-1 risk patients, achieved treatment success; reductions in spleen length and symptoms were observed in all risk groups. The majority of patients (66.7%) experienced ≥ 50% reductions from baseline in spleen length at any time. Improvements in MF-SAF TSS were seen in 80.0%, 72.7%, and 72.2% of intermediate-1, intermediate-2, and high-risk patients, respectively. Consistent with other studies of ruxolitinib, the most common haematological adverse events were anaemia and thrombocytopenia. Results indicate that most patients with myelofibrosis, including intermediate-1 risk patients, may benefit from ruxolitinib treatment.

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          Author and article information

          Br. J. Haematol.
          British journal of haematology
          Jul 2015
          : 170
          : 1
          [1 ] MRC Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, UK.
          [2 ] Imperial College Healthcare NHS Trust, Hammersmith Hospital, London, UK.
          [3 ] Department of Haematology, Cardiff University, Heath Park, Cardiff, UK.
          [4 ] Leicester Royal Infirmary, Leicester, UK.
          [5 ] The Royal Bournemouth and Christchurch Hospitals NHS Foundation Trust, Bournemouth, UK.
          [6 ] Department of Haematology, Western General Hospital, Edinburgh, UK.
          [7 ] Department of Haematology, Manchester Royal Infirmary, Manchester, UK.
          [8 ] Hull and East Yorkshire Hospitals NHS Trust, Hull, UK.
          [9 ] Department of Haematology, Royal Liverpool University Hospital, Liverpool, UK.
          [10 ] Novartis Pharmaceuticals UK, Frimley, UK.
          [11 ] Guy's and St. Thomas' NHS Foundation Trust, London, UK.
          © 2015 John Wiley & Sons Ltd.


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