- Non-JAK inhibitor Therapies for Myelofibrosis
- Risk Stratification and Prognostication
- Transplantation, COVID-19 and Biology Insights
- Novel Therapies for MPNs and JAK inhibitors for Myelofibrosis
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.
634. Myeloproliferative Syndromes: Clinical and Epidemiological: Risk Stratification and Prognostication
Khalid Abu-Zeinah, Khalil Saadeh, Richard T. Silver, et al.
An unacceptably high excess mortality has been observed in MPN patients under the age of 60. It is higher than in patients over 60 years of age.
Tiziano Barbui, Arianna Ghirardi, Alessandra Carobbio, et al.
The IPSS score can be useful in conjunction with the JAK2 mutation in addition to assessing the risk of survival. It helps to identify patients at vascular risk and suggest suitable antithrombotic prophylaxis.
Giuseppe Gaetano Loscocco, Paola Guglielmelli, Carmela Mannarelli, et al.
AT and VT are associated with unique risk factors in patients with PV. This is illustrated by data with JAK2VF VAF as a strong independent predictor of future venous thrombosis in PV associated with the previous history of venous events.
Ayalew Tefferi, Giuseppe Gaetano Loscocco, Faiqa Farrukh, et al.
Elevated ANC and decreased ALC are age-independent risk factors for survival in ET. The present study has identified this and thus enables the development of a globally applicable, easy-to-use “triple-A” risk model based on age, ANC, and ALC. Decreased ALC also predicted fibrotic and leukemic progression. The observations of the study authors suggest a potential value for immune profiling as an additional prognostic tool in MPN.
Aaron T. Gerds, Ruben A. Mesa, John M. Burke, et al.
Increased HCT levels (> 45%) as well as leukocytes (> 11 × 109 / l) and PLT numbers (> 400 × 109 / l)) were each associated with an increased risk of TE. For more info see abstract.
Douglas Tremblay, Andrew Srisuwananukorn, Lukas Ronner, et al.
For more info see abstract.
634. Myeloproliferative Syndromes: Clinical and Epidemiological: Transplantation, COVID-19 and Biology Insights
Derek W Brown, Youjin Wang, Andrew St. Martin, et al.
See abstract for more info.
Nico Gagelmann, Anita Badbaran, Markus Ditschkowski, et al.
See abstract for more info.
Tiziano Barbui, Alessandra Iurlo, Arianna Masciulli, et al.
Ruxolitinib discontinuation is still a risk factor for a dismal outcome. See abstract for more info.
Joan How, Kathleen M.E. Gallagher, Yiwen Liu, et al.
See abstract for more info.
Lin-Pierre Zhao, Marine Cazaux, Nabih Maslah, et al.
See abstract for more info.
Michael W. Deininger, Daniel J. DeAngelo, Deepti H. Radia, et al.
See abstract for more info.
634. Myeloproliferative Syndromes: Clinical and Epidemiological: Novel Therapies for MPNs and JAK inhibitors for Myelofibrosis
Jason Gotlib, Jean-Jacques Kiladjian, Alessandro Vannucchi, et al.
According to the study authors, the results of the study suggest that pemigatinib is a long-term treatment option for patients with MLNFGFR1. This affects those who are not eligible for HSCT. Pemigatinib could also facilitate the transition to HSCT if the indication for treatment exists.
Francesca Palandri, Nicola Vianelli, David M Ross, et al.
Bomedemstat improves symptoms, is well tolerated, and reduces both platelet and white blood cell counts while maintaining hemoglobin levels. After six weeks, four out of five patients achieved complete remission of the peripheral blood count in the absence of disease progression.
Yi Zhang, Hu Zhou, Zhongxing Jiang, et al.
Jaktinib was well tolerated and safe in Chinese MF patients. With 100 mg BID, a significant reduction in the volume of the spleen and the burden of constitutional symptoms was achieved. There was also evidence of an improvement in red cell transfusion dependence.
Ronald Hoffman, Marina Kremyanskaya, Yelena Ginzburg, et al.
See abstract for more info.
Vikas Gupta, Abdulraheem Yacoub, Srdan Verstovsek, et al.
Fedratinb is generally well tolerated. Early GI prophylaxis can reduce the frequency and severity of gastrointestinal side effects. The thiamine level should be determined before the start of treatment and monitored regularly during treatment.
Yelena Ginzburg, Kamini Kirubamoorthy, Sinari Salleh, et al.
In order to quickly achieve a target hematocrit value below 45% in all PV patients without a phlebotomy, induction therapy with rusfertide administration twice a week has proven to be effective. It was successfully maintained with weekly rusfertide. Injections of rusfertide twice a week to rapidly lower the hematocrit was safe and well-tolerated.