Citations

11005 Background: Pazopanib is approved for soft tissue sarcoma pts after failure of other therapy, but there are few subtype-specific data regarding kinase inhibitor activity. We report on a single arm, phase II trial of REGO in advanced EWS. Methods: EWS pts (age > 18, ECOG 0-2, good organ function) who had at least 1 line of therapy and had PD within 6 mo were eligible. Prior oral kinase inhibitors were not allowed. Initial REGO dose was 160 mg PO QD x21 q28d. Dose reductions were employed for toxicity and AEs. The primary endpoint was PFS at 8 weeks (PFS8w) employing RECIST 1.1. Sample size of 30 allowed determination of the difference between PFS8w of 50% vs 25% with alpha = 0.05 and power of 91%. Results: 30 pts (median age 32, range 19-65; M/F = 20/10; ECOG 0/1/2 = 16/13/1; bone, 12; soft tissue, 18; median prior treatments 5, range 1-10) enrolled at 14 US sites (09/2014-03/2016). Most common grade (G3) toxicities were hypophosphatemia (6), hypertension (2), high ALT (2) and 1 each: fatigue, abd pain, diarrhea, hypokalemia, oral mucositis, neutropenia and rash; no G4 toxicities were noted. 13 pts required ≥1 dose reduction, most commonly hypophosphatemia (n = 7); 2 stopped REGO for toxicity. There was 1 death in the 30 day post study period, not REGO related. Median dose at study end: 140 mg (3.5 tabs, range 80-160 mg) 3 wks on/1wk off. 18/30 pts were without PD at 8 wks. Median PFS: 3.6 mo (95%CI 2.8-3.8 mo). PFS8w by KM was 73% (95%CI 57-89%). Best responses: PR/SD/PD/not evaluable of 3/18/7/2, for RECIST RR 10%. Two pts with PR had EWSR1 translocation by FISH; a third had CIC-DUX4. Median duration of response: 5.5 mo (95%CI 2.9-8.0). Median OS is not reached. Conclusions: The substudy met its primary endpoint. REGO toxicity was similar to that seen previously. Enrollment continues in LPS and OGS cohorts, and is being expanded to further study variant EWS without EWSR1-FLI1 fusion. Study of the existing tissue may elucidate which EWS patients may benefit from REGO. Clinical trial information: NCT02048371.

e21704 Background: Patients with myeloproliferative neoplasms (MPN) have difficult symptom burden (i.e. fatigue, pruritus, night sweats) not resolved by current medical therapies including jak inhibitor therapy. We hypothesize that on-line (at home) yoga might alleviate MPN symptoms and complement medical therapy. Methods: MPN patients, recruited by social media, were asked to complete 60 min/week of online yoga (designed for MPN patients) for 12 weeks and completed online self-report surveys (weeks 0,7,12 and 16) on participation (Qualtrics), disease features, and symptom burden (MPN Symptom Assessment Form; NIH PROMIS measures of pain, anxiety, depression, sleep, and sexual function). Results: Two hundred and forty-four MPN patients completed the eligibility survey, 134 were eligible, 55 completed the informed consent, and 38 MPN patients completed the 12-week intervention. The majority of participants were diagnosed with either PV (n = 16) or ET (n = 16), with MF being less common (n = 6). Approximately 37% (n = 14) of study participants averaged ≥60 min/week of yoga (i.e., demand). Yoga participation averaged 50.8±36.2 min/week. Overall, 68% of participants were satisfied with online yoga and 75% felt that is was helpful for coping with MPN-related symptoms (i.e., acceptability, practicality). Additionally, 75% of participants felt safe from injury while participating in online yoga. Only one adverse event was reported (irritated enlarged spleen). From week 0 to week 12 (n = 38), there were significant improvements in total symptom burden (effect size [ES] in standard deviation units = -0.36, p = 0.004), anxiety (ES = -0.67, p = 0.002), depression (ES = -0.41, p = 0.049), sleep (ES = -0.58, p < 0.001), and fatigue (ES = -0.33, p = 0.04). These improvements remained significant at follow-up week 16 (n = 28) for all outcome measures with a trend for maintained fatigue improvement (ES = -0.34, p = 0.06) (see Table 1). There were no significant differences in outcomes between those that averaged < 60 min/week of yoga compared to those that averaged ≥60 min/week of yoga. Conclusions: A 12-week online yoga intervention is feasible (i.e., accepted, practical) for MPN patients and merits a randomized trial as complementary therapy.

e21684 Background: Pharmacologic therapy stabilizes hematologic counts and splenomegaly in myeloproliferative neoplasms (MPN), however only partial symptom improvement typically occurs. Evidenced-based supportive care interventions are needed, however data is limited in patients with MPNs. Methods: Patients were recruited internationally via social media. Informed consent and online self-report surveys (Qualtrics) were completed capturing patient demographics, disease specific data, supportive care utilization, and symptom burden via MPN-SAF TSS. ANOVA, chi square tests, and Wilcoxon rank sum tests methods were applied. Results: Patients:1087 patient surveys were consented. Of these, 858 had 10 or more responses. There were 338 essential thrombocytosis (ET), 188 myelofibrosis (MF), 315 polycythemia vera (PV), and 17 other. In MF: DIPSS risk categories included low (8%), Int-1 (19%), Int-2 (29%), high (12%), and unknown (32%). Supportive care:The most frequently utilized therapies were aerobic activity in 442 patients (51.5%), massage in 244 (28.4%), yoga in 220(25.6%), nutrition in 216 (25.1%), and strength training in 204 (23.7%). Symptom association: Among all respondents, aerobic activity (p=<0.001), massage (p=<0.001), yoga (p=0.02), and strength training (p=<0.001) was associated with lower symptom burden by the MPN-SAF TSS however, nutrition was not (p=0.10). In ET and PV subgroups, yoga intervention was no longer statistically significant (p=0.35, p=0.14). In the MF subgroup, no intervention was found to be significant in any risk category. Conclusions: Supportive care with aerobic activity, massage, yoga, and strength training is associated with improved symptom burden in patients with MPN. Further studies are needed to understand the benefits of such therapies. [Table: see text]