Review
Oncology
Sherrie Mikhaeel, Ehab Atallah
Summary: Tyrosine kinase inhibitor (TKI) discontinuation, also known as treatment-free remission (TFR), is an important goal in the therapy of chronic myeloid leukemia (CML) due to the impact of TKI therapy on quality of life, long-term side effects, and financial burden. TKI discontinuation is particularly crucial for young CML patients as it affects their growth and development. Multiple studies have demonstrated the safety and feasibility of attempting TKI discontinuation in patients with sustained deep molecular remission.
CLINICAL LYMPHOMA MYELOMA & LEUKEMIA
(2023)
Review
Oncology
Theresa Steeb, Anja Wessely, Anne Petzold, Christoph Kohl, Michael Erdmann, Carola Berking, Markus V. Heppt
Summary: The study summarized the efficacy and safety of c-Kit inhibitors for unresectable or metastatic mucosal, acral or chronically sun-damaged melanoma. Results showed that c-Kit inhibitors are a valuable treatment option for patients with KIT mutant melanoma, with nilotinib having the highest objective response rate.
EUROPEAN JOURNAL OF CANCER
(2021)
Review
Oncology
Kota Yoshifuji, Koji Sasaki
Summary: The prognosis of CML-CP has significantly improved with the introduction of imatinib. Besides imatinib, other TKIs and a novel drug asciminib are now available. Long-term TKI therapy requires individualized selection based on toxicity profile to minimize chronic toxicity and the risk of adverse events. This review summarizes the characteristics and adverse event profile of each TKI and discusses future perspectives in the treatment of CML-CP.
FRONTIERS IN ONCOLOGY
(2022)
Article
Mathematical & Computational Biology
Yuji Okamoto, Mitsuhito Hirano, Kai Morino, Masashi K. Kajita, Shinji Nakaoka, Mayuko Tsuda, Kei-ji Sugimoto, Shigehisa Tamaki, Junichi Hisatake, Hisayuki Yokoyama, Tadahiko Igarashi, Atsushi Shinagawa, Takeaki Sugawara, Satoru Hara, Kazuhisa Fujikawa, Seiichi Shimizu, Toshiaki Yujiri, Hisashi Wakita, Kaichi Nishiwaki, Arinobu Tojo, Kazuyuki Aihara
Summary: This study successfully classified CML patients with different nilotinib responses by using a dynamical model and laboratory findings. A method was proposed to identify patients who failed to achieve a treatment goal based on data collected at three initial time points only.
NPJ SYSTEMS BIOLOGY AND APPLICATIONS
(2022)
Article
Hematology
Fadi G. Haddad, Koji Sasaki, Ghayas C. Issa, Guillermo Garcia-Manero, Farhad Ravandi, Tapan Kadia, Jorge Cortes, Marina Konopleva, Naveen Pemmaraju, Yesid Alvarado, Musa Yilmaz, Gautam Borthakur, Courtney DiNardo, Nitin Jain, Naval Daver, Nicholas J. Short, Elias Jabbour, Hagop Kantarjian
Summary: This study evaluated the outcome of TKI discontinuation in CML patients and identified factors associated with success rates of treatment-free remission (TFR). The study found that TFR is safe and feasible in patients with Ph-positive CML on TKI therapy. Achieving MR4 or MR4.5 for at least 5 years was correlated with a better outcome.
AMERICAN JOURNAL OF HEMATOLOGY
(2022)
Article
Pharmacology & Pharmacy
R. Ciftciler, I. C. Haznedaroglu
Summary: Philadelphia (Ph*)/BCR-A BL1-positive chronic myeloid leukemia (CML) is a neoplastic hematologic disorder that can be functionally cured using tyrosine kinase inhibitor (TKI) drugs. Factors such as individual characteristics, drug compliance, lifestyle preferences, comorbidities, and physician experience should be considered when selecting the appropriate first-line TKI for newly diagnosed CML patients.
EUROPEAN REVIEW FOR MEDICAL AND PHARMACOLOGICAL SCIENCES
(2021)
Article
Medicine, Research & Experimental
Massimo Breccia, Francesca Chiodi, Aurelio Pio Nardozza, Diletta Valsecchi, Valentina Perrone, Diego Sangiorgi, Elisa Giacomini, Maria Chiara Rendace, Paola Coco, Eleonora Premoli, Luca Degli Esposti
Summary: This study analyzed the economic burden for managing patients with chronic myeloid leukemia (CML) in 2nd or > 3rd lines with tyrosine kinase inhibitors (TKIs) in Italian clinical practice. The results showed that as the treatment lines increased, healthcare consumption and costs for CML patients also increased, with hospitalizations accounting for about half of the total expenditure.
ADVANCES IN THERAPY
(2023)
Article
Hematology
Yves Chalandon, Giulia Sbianchi, Luuk Gras, Linda Koster, Jane Apperley, Jenny Byrne, Urpu Salmenniemi, Henrik Sengeloev, Mahmoud Aljurf, Grzegorz Helbig, Francesca Kinsella, Goda Choi, Peter Remenyi, John A. Snowden, Marie Robin, Stig Lenhoff, Stephan Mielke, Jakob Passweg, Annoek E. C. Broers, Nicolaus Kroeger, Zeynep Arzu Yegin, Sen Mui Tan, Patrick J. Hayden, Donal P. McLornan, Ibrahim Yakoub-Agha
Summary: With the introduction of TKI, the number of allo-HCT for CP CML patients has decreased significantly. The use of 1, 2, or 3 TKI prior to allo-HCT does not affect the overall survival, progression-free survival, relapse incidence, or non-relapse mortality. However, the disease stage and KPS score significantly influence the survival and progression-free survival, emphasizing the importance of careful candidate selection and performing allo-HCT during CP1 phase.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Review
Oncology
Takaaki Ono
Summary: This review discusses the importance of selecting the optimal BCR-ABL1 tyrosine kinase inhibitors (TKIs) for newly diagnosed CML-CP patients, and how individualized treatment choices can be made based on factors such as patient background, treatment goals, and cost.
Article
Hematology
Sean Tracy, Hrishi Venkatesh, Can Hekim, Lynn M. Heltemes-Harris, Todd P. Knutson, Veronika Bachanova, Michael A. Farrar
Summary: Research on acute lymphoblastic leukemia patients has shown that CD4(+) T cell exhaustion may be a key factor in relapse. Combined treatment with tyrosine kinase inhibitors and anti-PD-L1 significantly improves patients’ survival rates. Depleting CD4(+) T cells prior to therapy may weaken the immune response, while treatment with anti-PD-L1 helps activate the anti-leukemia response.
Article
Economics
B. Muresan, C. Mamolo, J. C. Cappelleri, M. J. Postma, B. Heeg
Summary: The study found that compared with dasatinib and nilotinib, bosutinib may represent good value for money in treating 2L CP-CML patients.
APPLIED HEALTH ECONOMICS AND HEALTH POLICY
(2021)
Review
Medicine, General & Internal
Alessandra Iurlo, Daniele Cattaneo, Cristina Bucelli, Massimo Breccia
Summary: The use of TKI in CML patients has brought significant changes to treatment strategies. TKI dose modifications have various applications in different scenarios, aiming not only to improve patient's quality of life but also to achieve and maintain treatment responses.
JOURNAL OF CLINICAL MEDICINE
(2021)
Editorial Material
Cardiac & Cardiovascular Systems
Zeeshan Hussain, Lauren Baldassarre, Susan Dent, Neal L. Weintraub, Avirup Guha
Summary: This editorial summarizes a nationwide population-based study in Taiwan on cardiovascular events in patients with chronic myeloid leukemia treated with tyrosine kinase inhibitors.
EUROPEAN JOURNAL OF PREVENTIVE CARDIOLOGY
(2022)
Article
Hematology
Hagop M. Kantarjian, Mary Alma Welch, Elias Jabbour
Summary: This article analyzes the risks and benefits of established treatment practices for BCR::ABL1 tyrosine kinase inhibitors (TKIs) in chronic myeloid leukaemia and suggests that lower doses of TKIs can achieve similar treatment efficacy with less toxicity, better treatment compliance, and reduced cost of care. It also highlights that changing TKIs might not be necessary for patients who do not have an early molecular response, particularly for second-generation TKIs. The availability of generic TKIs would provide an effective, safe, and affordable therapy for the majority of chronic myeloid leukaemia patients. Any TKI costing more than $30,000-40,000 per year should be evaluated in relation to alternative modalities.
LANCET HAEMATOLOGY
(2023)
Article
Hematology
Hagop M. Kantarjian, Elias Jabbour, Michael Deininger, Elisabetta Abruzzese, Jane Apperley, Jorge Cortes, Charles Chuah, Daniel J. DeAngelo, John DiPersio, Andreas Hochhaus, Jeffrey Lipton, Franck E. Nicolini, Javier Pinilla-Ibarz, Delphine Rea, Gianantonio Rosti, Philippe Rousselot, Neil P. Shah, Moshe Talpaz, Shouryadeep Srivastava, Xiaowei Ren, Michael Mauro
Summary: Ponatinib has demonstrated deep and durable responses in patients with chronic-phase chronic myeloid leukemia, especially in those resistant to second-generation TKI treatment, and has shown good safety and survival outcomes.
AMERICAN JOURNAL OF HEMATOLOGY
(2022)
Letter
Pharmacology & Pharmacy
Corine Ekhart, Maja Matic, Agnes Kant, Laure Elens, Eugene van Puijenbroek, Ron van Schaik
Article
Biochemistry & Molecular Biology
Shana Claeys, Geertrui Denecker, Kaat Durinck, Bieke Decaesteker, Liselot M. Mus, Siebe Loontiens, Suzanne Vanhauwaert, Kristina Althoff, Caroline Wigerup, Daniel Bexell, Emmy Dolman, Kai-Oliver Henrich, Lea Wehrmann, Ellen M. Westerhout, Jean-Baptiste Demoulin, Candy Kumps, Tom Van Maerken, Genevieve Laureys, Christophe Van Neste, Bram De Wilde, Olivier De Wever, Frank Westermann, Rogier Versteeg, Jan J. Molenaar, Sven Pahlman, Johannes H. Schulte, Katleen De Preter, Frank Speleman
Article
Dermatology
Guillaume Dachy, Ronald R. de Krijger, Sylvie Fraitag, Ivan Theate, Benedicte Brichard, Suma B. Hoffman, Louis Libbrecht, Florence A. Arts, Pascal Brouillard, Miikka Vikkula, Nisha Limaye, Jean-Baptiste Demoulin
Article
Oncology
Simone Hettmer, Guillaume Dachy, Guido Seitz, Abbas Agaimy, Catriona Duncan, Marjolijn Jongmans, Steffen Hirsch, Iris Kventsel, Uwe Kordes, Ronald R. de Krijger, Markus Metzler, Orli Michaeli, Karolina Nemes, Anna Poluha, Tim Ripperger, Alexandra Russo, Stephanie Smetsers, Monika Sparber-Sauer, Eveline Stutz, Franck Bourdeaut, Christian P. Kratz, Jean-Baptiste Demoulin
Summary: Infantile myofibromatosis (IM) presents a wide clinical spectrum, ranging from inconspicuous solitary soft tissue nodules to life-threatening complications. Familial IM follows an autosomal dominant mode of inheritance and is linked to PDGFRB germline variants. Mutant receptors remain sensitive to tyrosine kinase inhibitors and require a second hit for full activation.
Letter
Dermatology
A. Onoufriadis, B. Boulouadnine, G. Dachy, T. Higashino, H. Y. Huang, C. K. Hsu, M. A. Simpson, K. Bork, J. B. Demoulin, J. A. McGrath
BRITISH JOURNAL OF DERMATOLOGY
(2021)
Review
Biochemistry & Molecular Biology
Emilie Guerit, Florence Arts, Guillaume Dachy, Boutaina Boulouadnine, Jean-Baptiste Demoulin
Summary: PDGFRA and PDGFRB are classical proto-oncogenes encoding receptor tyrosine kinases, with mutations linked to various tumors and diseases. Research has validated the effectiveness of tyrosine kinase inhibitors targeting PDGF receptors, such as imatinib, as a treatment for certain conditions.
CELLULAR AND MOLECULAR LIFE SCIENCES
(2021)
Article
Geriatrics & Gerontology
Morgane M. Thibaut, Martina Sboarina, Martin Roumain, Sarah A. Potgens, Audrey M. Neyrinck, Florence Destree, Justine Gillard, Isabelle A. Leclercq, Guillaume Dachy, Jean-Baptiste Demoulin, Anne Tailleux, Sophie Lestavel, Marialetizia Rastelli, Amandine Everard, Patrice D. Cani, Paolo E. Porporato, Audrey Loumaye, Jean-Paul Thissen, Giulio G. Muccioli, Nathalie M. Delzenne, Laure B. Bindels
Summary: This study identified alterations in bile acid metabolism and hepatobiliary secretion in cancer cachexia, with an increase in systemic inflammation contributing to impairments in the hepatobiliary transport system. Targeting bile acids using cholestyramine reduced hepatic inflammation, while reducing interleukin 6 levels counteracted changes in gene expression related to hepatobiliary transport, bile acid synthesis, and inflammation. Serum bile acid levels were increased in cachectic cancer patients and strongly correlated with systemic inflammation.
JOURNAL OF CACHEXIA SARCOPENIA AND MUSCLE
(2021)
Review
Cardiac & Cardiovascular Systems
Alexandre Persu, Piotr Dobrowolski, Heather L. Gornik, Jeffrey W. Olin, David Adlam, Michel Azizi, Pierre Boutouyrie, Rosa Maria Bruno, Marion Boulanger, Jean-Baptiste Demoulin, Santhi K. Ganesh, Tomasz J. Guzik, Magdalena Januszewicz, Jason C. Kovacic, Mariusz Kruk, Peter de Leeuw, Bart L. Loeys, Marco Pappaccogli, Melanie H. A. M. Perik, Emmanuel Touze, Patricia Van der Niepen, Daan J. L. Van Twist, Ewa Warchol-Celinska, Aleksander Prejbisz, Andrzej Januszewicz
Summary: Fibromuscular dysplasia (FMD) is a non-atherosclerotic vascular disease that predominantly affects women. Its exact cause is still poorly understood, but it is likely to involve a combination of genetic and environmental factors, leading to arterial fibrosis, cellular hyperplasia, and distortion of the arterial wall architecture. FMD can affect various arteries throughout the body, not just the renal and cerebrovascular arteries.
CARDIOVASCULAR RESEARCH
(2022)
Article
Biochemistry & Molecular Biology
Emeline Bollaert, Melissa Claus, Virginie Vandewalle, Sandrine Lenglez, Ahmed Essaghir, Jean-Baptiste Demoulin, Violaine Havelange
Summary: The study found that miR-15a-5p induces chemoresistance in AML cells by inhibiting daunorubicin-induced autophagy, suggesting that this miRNA could be a promising therapeutic target for chemoresistant AML patients.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Cell Biology
Guillaume Dachy, Sylvie Fraitag, Boutaina Boulouadnine, Sabine Cordi, Jean-Baptiste Demoulin
Summary: The study identified different gene fusions in myofibroma patients, including a novel COL4A1-VEGFD fusion, which provides new insights for the diagnosis and treatment of myofibroma.
JOURNAL OF CELLULAR AND MOLECULAR MEDICINE
(2021)
Article
Cell Biology
Audrey Nedelec, Emilie M. Guerit, Guillaume Dachy, Sandrine Lenglez, Lok San Wong, Florence A. Arts, Jean-Baptiste Demoulin
Summary: Penttinen syndrome is a rare progeroid disorder caused by mutations in the PDGF receptor beta gene. This study demonstrates that the PDGFRB p.Val665Ala variant has unique features at the molecular level, which may be associated with the symptoms of Penttinen syndrome.
JOURNAL OF CELLULAR AND MOLECULAR MEDICINE
(2022)
Article
Virology
Bradley Ward, Jean Cyr Yombi, Jean-Luc Balligand, Patrice D. Cani, Jean-Francois Collet, Julien de Greef, Joseph P. Dewulf, Laurent Gatto, Vincent Haufroid, Sebastien Jodogne, Benoit Kabamba, Sebastien Pyr Dit Ruys, Didier Vertommen, Laure Elens, Leila Belkhir
Summary: This paper introduces the HYGIEIA project, which aims to study the molecular mechanisms and post-COVID conditions through a multi-omic approach and network medicine analysis. By analyzing high-throughput sequencing and mass-spectrometry data from multiple biological layers, meaningful discoveries are expected and can be translated into improvements in clinical practice.
Article
Infectious Diseases
Perrin Ngougni Pokem, Xavier Wittebole, Christine Collienne, Hector Rodriguez-Villalobos, Paul M. Tulkens, Laure Elens, Francoise Van Bambeke, Pierre-Francois Laterre
Summary: This study investigated the pharmacokinetics of temocillin in plasma and ascitic fluid, and performed dosing simulations. The current dosing regimen achieved the target concentration in plasma, but not in ascitic fluid. Adjusting the dosing regimen might allow for target concentration attainment in ascitic fluid, but further evaluation is needed for safety and efficacy.
Review
Biochemistry & Molecular Biology
Emeline Bollaert, Audrey de Rocca Serra, Jean-Baptiste Demoulin
CELLULAR AND MOLECULAR LIFE SCIENCES
(2019)
Article
Hematology
Anne-Laure Sennesael, Anne-Sophie Larock, Jonathan Douxfils, Laure Elens, Gabriel Stillemans, Martin Wiesen, Max Taubert, Jean-Michel Dogne, Anne Spinewine, Francois Mullier
THROMBOSIS JOURNAL
(2018)
