PB2235 SYMPTOM BURDEN AND SPLEEN SIZE RESPONSE WITH RUXOLUTINIB IN A REAL‐LIFE TURKISH COHORT
Authors: Tuglular T. , Arikan F. , Ercan T. , Elibol T. , Okcu D. , Ipek Y. , Ozgumus T. , Sezgin A. , Yilmaz F. , Atagunduz I. , Toptas T. 
Published: June 2019

Background:

Myelofibrosis (MF) is a myeloproliferative disease that is characterized by variable life expectancy, cytopenia, splenomegaly, and constitutional symptoms. Although allogeneic stem cell transplantation can be a curative treatment, it carries a high risk of morbidity and mortality (1,2). Ruxolitinib, a JAK2 inhibitor, is a new treatment option which improves symptomatic splenomegaly in patients with intermediate and high risk myelofibrosis.

Aims:

In this retrospective study we documented our real life clinical experience with ruxolitinib in MF patients.

Methods:

We reviewed the data of 53 patients with MF who were treated with ruxolitinib. Primary endpoint is to identify the improvement in spleen size and symptoms of the patients. Secondary endpoint was to determine overall survival of the patients, treatment safety and tolerability. Spleen size was measured by using ultrasound examination. Myeloproliferative Neoplasm 10 (MPN‐10) disease‐specific questionnaire was used to evaluate the symptoms and quality of life.

Results:

Fifty three patients with a mean age of 63.5 years were evaluated. Mean follow‐up time on ruxolitinib treatment was 18.9 ± 17.3 months (range 1‐69 months). DIPSS plus score was Int‐1 in 33 patients (62.3%), Int‐2 in 16 patients (30.2%), high risk in 4 patients (7.5%). Baseline demographic data of the patients were summarized in Table 1. The median subcostal spleen size decreased from 80 (34‐272) mm to 49 (0‐210) mm in 28 (52%) patients. Median spleen size reduction was 45% (10‐100%) in these patients. Spleen size decreased by 35% in 17 (32%) patients (Figure 1). In six patients (11 %), there were no change in spleen size. Spleen size increased 29.5% in 12 patients (1.5‐ 85.7%). Percentage of the patients whose spleen length did not change or increase were relatively high in post ET‐MF patients compared to patients with primary MF (66% vs. 34 %, respectively, P > 0.05). MPN‐10 reassessment data within the first 6‐12 months was documented in 15 patients and found to be improved in 10 of 15 patients (66%). Hematologic adverse events were found in %77.4 which includes 73.6% anemia, 28.3% thrombocytopenia. Grade 2‐3 anemia and grade 3 thrombocytopenia was seen in 50% and 13% of the patients, respectively. Non‐hematological adverse events were observed in 11.3% of patients. Overall survival of the cohort was 66.7 ± 46.3 (5‐206) months. A total of 10 patients died, in three of them the cause of death was leukemic transformation.

Summary/Conclusion:

Our results which are similar to previous studies show that ruxolitinib is associated with improvement in quality of life and splenomegaly (3). The most important hematological side effects are anemia and thrombocytopenia and non‐hematological side effects are acceptable. The main difference of our real‐life data from the previous randomized clinical studies is that the majority of our patients had intermediate‐I disease. In this real‐life data we showed that ruxolutinib is safe and effective in this group of patients.

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