Article
Oncology
Simone Claudiani, Clinton C. Mason, Dragana Milojkovic, Andrea Bianchi, Cristina Pellegrini, Antinisca Di Marco, Carme R. Fiol, Mark Robinson, Kanagaraju Ponnusamy, Katya Mokretar, Avirup Chowdhury, Michael Albert, Alistair G. Reid, Michael W. Deininger, Kikkeri Naresh, Jane F. Apperley, Jamshid S. Khorashad
Summary: By inhibiting the proteasome family, carfilzomib in combination with ruxolitinib enhances suppression of primary MF cells in vitro. This suggests a potential role for proteasome inhibitors in combination with ruxolitinib for management of MF patients.
Article
Oncology
Nicolaus Kroeger, Giulia Sbianchi, Tiarlan Sirait, Christine Wolschke, Dietrich Beelen, Jakob Passweg, Marie Robin, Radovan Vrhovac, Grzegorz Helbig, Katja Sockel, Eibhlin Conneally, Marie Therese Rubio, Yves Beguin, Juergen Finke, Paolo Bernasconi, Elena Morozova, Johannes Clausen, Peter von dem Borne, Nicolaas Schaap, Wilfried Schroyens, Francesca Patriarca, Nicola Di Renzo, Zeynep Arzu Yegin, Patrick Hayden, Donal McLornan, Ibrahim Yakoub-Agha
Summary: This study found that pretreatment with the JAK1/2 inhibitor ruxolitinib (RUX) and ongoing spleen response at the time of transplantation were associated with lower relapse risk and better 2-year event-free survival in myelofibrosis patients undergoing hematopoietic stem cell transplantation (HSCT). Age and HLA mismatch donor were significant factors affecting overall survival.
Review
Oncology
Srdan Verstovsek, Ruben A. Mesa, Robert A. Livingston, Wilson Hu, John Mascarenhas
Summary: Myelofibrosis is a chronic myeloproliferative neoplasm characterized by bone marrow fibrosis, anemia, extramedullary hematopoiesis, and splenomegaly. Ruxolitinib, an oral JAK1/JAK2 inhibitor, has been approved for the treatment of intermediate or high-risk MF patients. It remains the standard of care for higher-risk MF, and dose optimization and management are crucial for maximizing its benefits.
JOURNAL OF HEMATOLOGY & ONCOLOGY
(2023)
Article
Oncology
Vera Stoeva, Georgi Mihaylov, Konstantin Mitov, Guenka Petrova, Konstantin Tachkov
Summary: This study analyzed the therapeutic results and survival of patients with myelofibrosis treated with ruxolitinib compared to standard therapy. The results showed that patients treated with ruxolitinib exhibited improved clinical signs, lower symptom severity, and longer survival.
Article
Hematology
Vikas Gupta, John Mascarenhas, Marina Kremyanskaya, Raajit K. Rampal, Moshe Talpaz, Jean-Jacques Kiladjian, Alessandro M. Vannucchi, Srdan Verstovsek, Gozde Colak, Debarshi Dey, Claire Harrison
Summary: A study suggests that pelabresib in combination with ruxolitinib may have higher efficacy in treating JAKi treatment-naive MF patients compared to JAKi monotherapy, with significant improvements in spleen volume reduction and total symptom scores.
Article
Oncology
Tamara K. Moyo, Ashwin Kishtagari, Matthew T. Villaume, Brandon McMahon, Sanjay R. Mohan, Tess Stopczynski, Sheau-Chiann Chen, Run Fan, Yuankai Huo, Hyeonsoo Moon, Yucheng Tang, Cosmin A. Bejan, Merrida Childress, Ingrid Anderson, Kyle Rawling, Rhea M. Simons, Ashley Moncrief, Rebekah Caza, Laura Dugger, Aunshka Collins, Channing Dudley, P. Brent Ferrell, Michael Byrne, Stephen A. Strickland, Gregory D. Ayers, Bennett A. Landman, Emily F. Mason, Ruben A. Mesa, Jeanne M. Palmer, Laura C. Michaelis, Michael R. Savona
Summary: This study evaluated the safety and efficacy of a combination treatment of a selective PI3KS inhibitor, umbralisib, and a JAK inhibitor, ruxolitinib, for patients with MF. The results showed that the combination treatment was well tolerated and could potentially resensitize patients to ruxolitinib.
CLINICAL CANCER RESEARCH
(2023)
Review
Oncology
Francesco Passamonti, Florian H. Heidel, Rohan C. Parikh, Mayank Ajmera, Derek Tang, Jose Alberto Nadal, Keith L. Davis, Pranav Abraham
Summary: Real-world data analysis reveals the unmet need for more effective treatments for patients with myelofibrosis, especially those who failed ruxolitinib treatment.
Article
Chemistry, Medicinal
Vera Stoeva, Guenka Petrova, Konstantin Mitov, Konstantin Tachkov, Yuhei Nishimura
Summary: This study aimed to explore symptom severity and adherence to therapy for patients with myelofibrosis treated with ruxolitinib in Bulgaria. The results showed that ruxolitinib treatment decreased symptom severity and spleen size, and patients exhibited high adherence to therapy during the observation period.
Article
Oncology
Lucia Masarova, Prithviraj Bose, Naveen Pemmaraju, Naval Daver, Lingsha Zhou, Sherry Pierce, Hagop Kantarjian, Zeev Estrov, Srdan Verstovsek
Summary: The study analyzed the outcomes of 1412 patients with myelofibrosis based on bone marrow blasts and exposure to ruxolitinib (RUX). Patients with 5% to 9% blasts had adverse clinical course but benefited from RUX treatment.
CLINICAL LYMPHOMA MYELOMA & LEUKEMIA
(2021)
Article
Hematology
Naveen Pemmaraju, Jacqueline S. Garcia, Jalaja Potluri, Jason G. Harb, Yan Sun, Paul Jung, Qin Q. Qin, Srinivas K. Tantravahi, Srdan Verstovsek, Claire Harrison
Summary: The exploratory post-hoc biomarker analyses from cohort 1a of the REFINE trial showed that the addition of navitoclax to ruxolitinib led to clinically meaningful splenic responses in patients with myelofibrosis who were no longer benefiting from ruxolitinib monotherapy. Improvements in fibrosis or a reduction in variant allele frequency were associated with improved overall survival, suggesting potential disease modification. These findings highlight the therapeutic potential of combining navitoclax with ruxolitinib for patients with myelofibrosis who had disease progression or suboptimal response to ruxolitinib monotherapy.
LANCET HAEMATOLOGY
(2022)
Review
Hematology
Francesco Passamonti, Barbara Mora
Summary: The clinical phenotype of myelofibrosis is characterized by splenomegaly, symptomatology, blood cell alterations, and vascular complications. Diagnosis requires evaluations of blood counts, bone marrow morphology, genetic mutations, and disease history. Treatment options include anemia-oriented therapies and JAK inhibitors, with stem cell transplant being the only curative option. New paradigms for evaluating efficacy and translational studies show promise for future strategies in MF patients.
Review
Oncology
Lea Sureau, Corentin Orvain, Jean-Christophe Ianotto, Valerie Ugo, Jean-Jacques Kiladjian, Damien Luque Paz, Jeremie Riou
Summary: Myelofibrosis is a myeloproliferative neoplasm with constitutional symptoms, splenomegaly, and cytopenias. JAK inhibitors are effective in treatment, with momelotinib and fedratinib showing comparable efficacy to ruxolitinib in first-line therapy. Different JAK inhibitors have varying tolerances and effectiveness for myelofibrosis treatment, with some potential options beyond FDA-approved drugs.
BLOOD CANCER JOURNAL
(2021)
Article
Hematology
Haris Ali, Ni-Chun Tsai, Timothy Synold, Sally Mokhtari, Weimin Tsia, Joycelynne Palmer, Tracey Stiller, Monzr Al Malki, Ibrahim Aldoss, Amandeep Salhotra, Syed Rahmanuddin, Vinod Pullarkat, Ji-Lian Cai, Anthony Stein, Stephen J. Forman, Guido Marcucci, Matthew Mei, David S. Snyder, Ryotaro Nakamura
Summary: This study reports the safety and feasibility of peritransplantation ruxolitinib for myelofibrosis treatment. The results show that this treatment is safe and well tolerated at a specific dose, and has promising early efficacy in high-risk older patients with myelofibrosis.
Article
Hematology
Srdan Verstovsek, Shreekant Parasuraman, Jingbo Yu, Anne Shah, Shambhavi Kumar, Ann Xi, Claire Harrison
Summary: This retrospective analysis examined the real-world impact of the Janus kinase inhibitor ruxolitinib on overall survival in patients with myelofibrosis. The study found that regardless of exposure to ruxolitinib, the risk of mortality was significantly lower post-approval compared to preapproval, with a greater survival benefit observed in ruxolitinib-exposed patients. The findings support the clinical data of ruxolitinib in myelofibrosis by demonstrating a survival benefit in a real-world setting.
ANNALS OF HEMATOLOGY
(2022)
Review
Pharmacology & Pharmacy
Yuanyuan Ma, Zhiyuan Zhou, Guo-Yuan Yang, Jing Ding, Xin Wang
Summary: This review provides comprehensive information on the treatment of ischemic stroke with erythropoietin (EPO) and its derivatives. It covers the structure, function, pre-clinical studies, clinical trials, and therapeutic mechanisms of EPO, including its role in suppressing inflammation. The advanced studies on the therapy using EPO derivatives are also discussed. The findings of this review are valuable for basic researchers and clinicians in accelerating the clinical applications of EPO and its derivatives in ischemic stroke.
FRONTIERS IN PHARMACOLOGY
(2022)