634. Myeloproliferative Syndromes: Clinical and Epidemiological: Non-JAK inhibitor Therapies for Myelofibrosis
Harinder Gill, Abdulraheem Yacoub, Kristen M. Pettit, et al.
The once-daily monotherapy administration of bomedemstat has an acceptable tolerability profile without significant safety signals. It provides symptom relief in these patients with advanced disease, high mutation levels, and limited treatment options. The spleen volume decreased and the anemia improved. Additional data will be presented in the oral session.
Abdulraheem Yacoub, Mrinal M. Patnaik, Haris Ali, et al.
In high-risk myelofibrosis patients, TAG monotherapy demonstrates that the treatment is clinically effective and has a predictable and manageable safety profile. This also applies to patients refractory to JAKi and those with associated monocytosis, thrombocytopenia, advanced disease, and high-risk genetic characteristics.
Marina Kremyanskaya, John Mascarenhas, Francesca Palandri, et al.
Pelabresib monotherapy is generally well tolerated. It shows clinical activity signals in MF patients who are JAKi intolerant/refractory or not suitable for JAKi. It is also a group of patients who have limited treatment options and poor outcomes.
John Mascarenhas, Heidi E. Kosiorek, Rupali Bhave, et al.
The TGFβ1/3 protein trap AVID200 was well tolerated. However, the clinical response at therapy cycle 7 in this patient population with advanced MF was limited according to the IWG/MRI response criteria. Since AVID200 therapy leads to a significant reduction in serum TGFβ levels and an improvement in platelet count, it is believed that TGFβ1 plays a crucial role in MF. This leads to thrombocytopenia, which however can be reversed with AVID200 therapy. The authors conclude that AVID200 can be used in combination therapy approaches for thrombocytopenic MF patients.
Srinivas K. Tantravahi, Soo Jin Kim, Divya Sundar, et al.
Weekly monotherapy with per os Selinexor leads to a persistent splenic reaction in patients with JAKi-refractory MF. The treatment was well tolerated over time. Further dates will be presented in the oral session.
Prithviraj Bose, Lucia Masarova, Naveen Pemmaraju, et al.
Anemia of MPN-associated MF can be safely and effectively treated with Sotatercept. This applies to both non-TD and TD patients. The response rates are 30 percent when used alone and 32 percent when combined with a stable dose of Rux. All responses in the Rux cohort were observed in non-TD patients.