634. Myeloproliferative Syndromes: Clinical: Emerging and Novel Targeted Therapies

Conclusion cited from the abstract: Ropeg minimizes the occurrence of thromboembolic events in patients with PV over long-term treatment, without leukemogenic risk. In addition, we show for the first time in a randomized study that, in contrast to hydroxyurea, long-term ropeg treatment is capable of inducing deep molecular responses including CMR, which underscores its disease modifying potential. These results also suggest that selected patients could achieve operational cure (with both CHR and CMR) with ropeg, opening the way for treatment discontinuation.



Sara T Olalla Saad et al. 

Conclusion cited from the abstract: In this study, metformin showed to be a safe and well-tolerated drug. Our preliminary results demonstrated a trend in BM collagen reduction in PMF patients following metformin treatment. Downregulation of important genes associated with MPN phenotype was also noted. The trial is ongoing and these results will be validated at other time points for all subjects.



Conclusion cited from the abstract: In an older group of pts with 70% with ≥2 prior therapies (55% JAK inhibitor-exposed), median baseline platelet count of 51, ASXL1-mutated in 23%, we observed a 32% ORR. The median OS has not yet been reached. Oral SMAC mimetics may represent a possible option for older pts, those who have failed prior JAK inhibitor, and those with thrombocytopenia limiting entry onto other trials. Future directions include combination studies with hypomethylating agents and JAK inhibitors for pts with myeloid malignancies. This clinical trial is registered at ClinicalTrials.gov as NCT02098161.



Conclusion cited from the abstract: 

This is the first clinical study of an LSD1 inhibitor in patients with MPNs. Once-daily IMG-7289 was well-tolerated in a heterogeneous population of patients with advanced MF and limited therapeutic options. Despite under-dosing and slow dose escalation, IMG-7289 improved symptom burdens in most patients and modestly reduced spleen volumes in a subset of patients. The Phase 2b 24-week expansion study with more aggressive dosing aimed at preserving safety and enhancing efficacy is open for enrollment in the US, UK and EU.






Conclusion cited from the abstract: The initial results from this ongoing study suggest clinically significant activity of luspatercept in patients with MF-associated anemia, including those receiving concomitant ruxolitinib. A minority of AEs were grade 3–4 in severity, consistent with previous studies in MDS and beta-thalassemia.




Conclusion cited from the abstract: Tagraxofusp demonstrated single agent clinical activity, with a predictable and manageable safety profile, in patients with relapsed/refractory MF, including in patients with monocytosis, a poor prognostic factor and unmet medical need. Tagraxofusp may offer MF patients, and MF patients with monocytosis in particular, a novel treatment option. Enrollment continues, and updated trial data will be presented. This clinical trial is registered at clinicaltrials.gov: NCT02268253.