Article
Pharmacology & Pharmacy
Ling Yang, Pei Shu, Nan Wu, Mengyue Hu, Zhu Luo
Summary: The study aimed to investigate the pharmacokinetics, pharmacodynamics, safety and tolerability of FTP-198 in healthy subjects. The results showed that FTP-198 had good pharmacokinetic properties in healthy subjects, effectively inhibited the content of plasma lysophosphatidic acid (LPA) 18:2, and exhibited favorable safety and tolerability. These results support further subsequent clinical development of FTP-198 in IPF patients.
EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES
(2023)
Article
Pharmacology & Pharmacy
Jingrui Liu, Xiaoxue Zhu, Hong Zhang, Haijing Wei, Deming Yang, Zhongnan Xu, Dandan Huo, Xiaojiao Li, Yanhua Ding
Summary: TQ-F3083 showed good tolerability in healthy Chinese adults, with pharmacokinetic and pharmacodynamic characteristics supporting further evaluation in T2DM patients.
FRONTIERS IN PHARMACOLOGY
(2021)
Article
Microbiology
Sinenhlanhla Mtshali, Byron A. Jacobs
Summary: Physiologically based pharmacokinetic models have gained popularity in drug development and regulatory science. This study validates the model's effectiveness in predicting drug-drug interaction and the pharmacokinetics of antiretroviral and anti-TB drugs through mathematical solving and sensitivity analysis.
ANTIMICROBIAL AGENTS AND CHEMOTHERAPY
(2022)
Article
Immunology
Meghan E. Whalen, Richard Kajubi, Justin Goodwin, Francis Orukan, McKenzie Colt, Liusheng Huang, Kacey Richards, Kaicheng Wang, Fangyong Li, Norah Mwebaza, Francesca T. Aweeka, Sunil Parikh
Summary: Extending the duration of AL can increase drug exposure in young children, but it does not significantly reduce the risk of recurrent parasitemia in a high-transmission setting. Clinically, day 7 lumefantrine levels can be used as an indicator to predict the risk of recurrent parasitemia.
CLINICAL INFECTIOUS DISEASES
(2023)
Article
Oncology
David Van Mater, Sridharan Gururangan, Oren Becher, Olivia Campagne, Sarah Leary, Joanna J. Phillips, Jie Huang, Tong Lin, Tina Young Poussaint, Stewart Goldman, Patricia Baxter, Girish Dhall, Giles Robinson, Mariko DeWire-Schottmiller, Eugene Hwang, Clinton F. Stewart, Arzu Onar-Thomas, Ira J. Dunkel, Maryam Fouladi
Summary: The study found that palbociclib can be safely administered to children and adolescents with brain tumors, with a dosage of 75 mg/m(2) being optimal. However, no objective responses to palbociclib therapy were observed. Future studies will determine the best utilization of palbociclib in pediatric patients with brain tumors.
PEDIATRIC BLOOD & CANCER
(2021)
Article
Infectious Diseases
Pierre Chauvelot, Celine Dupieux-Chabert, Lelia Abad, Aubin Souche, Tristan Ferry, Jerome Josse, Frederic Laurent, Florent Valour
Summary: The study demonstrated that dalbavancin has the ability to effectively eradicate intraosteoblastic reservoir of Staphylococcus aureus in bone and joint infections. It showed significant reduction in bacterial inoculum at both low concentration and standard therapeutic doses, with similar efficacy to vancomycin but less efficient than rifampicin. Dalbavancin was found to be the only molecule significantly active at a low concentration.
JOURNAL OF ANTIMICROBIAL CHEMOTHERAPY
(2021)
Article
Endocrinology & Metabolism
Ki Young Huh, Jun Gi Hwang, Wonjung Shin, Seungjae Baek, JaeDuk Choi, Nora Lee, Young Min Cho, Howard Lee
Summary: HM15136, a novel long-acting glucagon analogue, was shown to be safe and well tolerated in doses ranging from 10-120 μg/kg in a randomized, double-blind, placebo-controlled study. It increased blood glucose levels and suppressed the secretion of endogenous glucagon.
DIABETES OBESITY & METABOLISM
(2022)
Article
Endocrinology & Metabolism
David Z. I. Cherney, Dick de Zeeuw, Hiddo J. L. Heerspink, Jose Cardona, Marc Desch, Arne Wenz, Friedrich Schulze, Masaomi Nangaku
Summary: The study aimed to assess the safety and efficacy of the NO-independent sGC activator BI 685509 in patients with diabetic kidney disease and albuminuria. The results showed that BI 685509 has potential in lowering UACR values in these patients.
DIABETES OBESITY & METABOLISM
(2023)
Article
Public, Environmental & Occupational Health
David S. Lawrence, Charles Muthoga, David B. Meya, Lillian Tugume, Darlisha Williams, Radha Rajasingham, David R. Boulware, Henry C. Mwandumba, Melanie Moyo, Eltas N. Dziwani, Hendramoorthy Maheswaran, Cecilia Kanyama, Mina C. Hosseinipour, Chimwemwe Chawinga, Graeme Meintjes, Charlotte Schutz, Kyla Comins, Funeka Bango, Conrad Muzoora, Samuel Jjunju, Edwin Nuwagira, Mosepele Mosepele, Tshepo Leeme, Chiratidzo E. Ndhlovu, Admire Hlupeni, Shepherd Shamu, Timothee Boyer-Chammard, Sile F. Molloy, Nabila Youssouf, Tao Chen, Tinevimbo Shiri, Shabbar Jaffar, Thomas S. Harrison, Joseph N. Jarvis, Louis W. Niessen
Summary: This cost-effectiveness analysis involving five countries found that a treatment regimen based on a single high dose of liposomal amphotericin B deoxycholate is cost-effective for HIV-associated cryptococcal meningitis and may even be cost-saving in real-world implementation.
LANCET GLOBAL HEALTH
(2022)
Article
Pharmacology & Pharmacy
Jinhong Wu, Yuan Han, Yu Lu, Yan Zhuang, Wenxian Li, Ji'e Jia
Summary: This study compared the pharmacokinetics and pharmacodynamics of dexmedetomidine administered intravenously, intranasally, and orally, and found that intravenous administration provided the best surgical field visibility. Intranasal administration was more suitable for tympanic membrane perforation repair compared to oral administration.
FRONTIERS IN PHARMACOLOGY
(2022)
Review
Pharmacology & Pharmacy
Li Zhou, Micaela B. Reddy, Rajendar K. Mittapalli, Jing Yang, Donghua Yin
Summary: Combination therapies are commonly evaluated in the clinical development of oncology investigational agents, and the design needs to consider factors such as drug interactions and benefit/risk to study participants. This report proposes application scenarios for different trial designs and emphasizes the importance of generating robust exposure-response data for optimal dose selection.
CLINICAL PHARMACOLOGY & THERAPEUTICS
(2023)
Article
Pharmacology & Pharmacy
Ana Alarcia-Lacalle, Helena Barrasa, Javier Maynar, Andres Canut-Blasco, Carmen Gomez-Gonzalez, Maria Angeles Solinis, Arantxazu Isla, Alicia Rodriguez-Gascon
Summary: This study developed a rapid, simple and reproducible HPLC-UV method for quantifying ceftaroline in plasma samples, which was accurate and stable, successfully applied in two critically ill patients.
Review
Infectious Diseases
Cornelia B. Landersdorfer, Amanda Gwee, Roger L. Nation
Summary: Traditionally, ongoing i.v. antibiotic therapy has been believed to be superior to early i.v. to oral switch for severe infections. However, this belief may not be based on robust data and contemporary clinical studies. This review examines the rationale for an early switch and discusses barriers and perceptions related to this practice.
CLINICAL MICROBIOLOGY AND INFECTION
(2023)
Article
Oncology
Daisuke Shinoda, Yaeko Nakajima-Takagi, Motohiko Oshima, Shuhei Koide, Kazumasa Aoyama, Atsunori Saraya, Hironori Harada, Bahityar Rahmutulla, Atsushi Kaneda, Kiyoshi Yamaguchi, Yoichi Furukawa, Haruhiko Koseki, Kazuya Shimoda, Tomoaki Tanaka, Goro Sashida, Atsushi Iwama
Summary: The insufficiency of PRC1.1, a component of polycomb repressive complex 1, promotes the development of myelofibrosis and acts as a tumor suppressor in this process. This impact is distinct from the effect of PRC2 insufficiency on the pathogenesis of myelofibrosis.
Article
Hematology
Julie Kanter, R. Clark Brown, Cynthia Norris, Santosh M. Nair, Abdullah Kutlar, Deepa Manwani, Nirmish Shah, Chiaki Tanaka, Shankaranand Bodla, Gessami Sanchez-Olle, Urs Albers, Darla Liles
Summary: This study analyzed the pharmacokinetics, pharmacodynamics, safety, and efficacy of crizanlizumab in patients with sickle cell disease. The results demonstrated the sustained efficacy and long-term safety of the drug in treating sickle cell disease.
Article
Hematology
Marco Pizzi, Gianni Binotto, Giulia Rigoni Savioli, Angelo Paolo Dei Tos, Attilio Orazi
Summary: Bone marrow studies are essential for the diagnosis of haematological disorders and can be traced back to the work of Giovanni Ghedini. His research was based on his clinical experience and the scientific developments of his time.
BRITISH JOURNAL OF HAEMATOLOGY
(2022)
Article
Hematology
Saar Gill, Vanessa Vides, Noelle Frey, Elizabeth O. Hexner, Susan Metzger, Megan O'Brien, Wei-Ting Hwang, Jennifer L. Brogdon, Megan M. Davis, Joseph A. Fraietta, Avery L. Gaymon, Whitney L. Gladney, Simon F. Lacey, Anne Lamontagne, Anthony R. Mato, Marcela Maus, J. Joseph Melenhorst, Edward Pequignot, Marco Ruella, Maksim Shestov, John C. Byrd, Stephen J. Schuster, Donald L. Siegel, Bruce L. Levine, Carl H. June, David L. Porter
Summary: Adding autologous anti-CD19 humanized binding domain T cells (huCART-19) to ibrutinib treatment in patients with chronic lymphocytic leukemia (CLL) who did not achieve complete remission (CR) despite >= 6 months of ibrutinib resulted in high rates of deep and durable remissions.
Review
Hematology
Jurgen Thiele, Hans Michael Kvasnicka, Attilio Orazi, Umberto Gianelli, Naseema Gangat, Alessandro M. Vannucchi, Tiziano Barbui, Daniel A. Arber, Ayalew Tefferi
Summary: A group of international experts met to update the World Health Organization classification system for hematopoietic tumors and introduced the new International Consensus Classification (ICC) for Myeloid Neoplasms and Acute Leukemias. The focus of this review is on the ICC-2022 category of JAK2 mutation-prevalent myeloproliferative neoplasms (MPNs) and the importance of bone marrow morphology and genetic markers in disease classification and diagnostics.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Review
Pathology
Roos J. Leguit, Sa A. Wang, Tracy George, Alexandar Tzankov, Attilio Orazi
Summary: Mastocytosis is a neoplasm characterized by clonal proliferation of mast cells, which can accumulate in the skin and multiple organs, leading to various clinical presentations. According to the 2022 international consensus classification, mastocytosis can be divided into a benign form confined to the skin and a malignant form with systemic involvement.
Correction
Hematology
Juergen Thiele, Hans Michael Kvasnicka, Attilio Orazi, Umberto Gianelli, Naseema Gangat, Alessandrom M. Vannucchi, Tiziano Barbui, Daniel A. Arber, Ayalew Tefferi
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Editorial Material
Hematology
Daniel A. A. Arber, Robert P. P. Hasserjian, Attilio Orazi
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Review
Pathology
Sonam Prakash, Daniel A. Arber, Carlos Bueso-Ramos, Robert P. Hasserjian, Attilio Orazi
Summary: The MDS/MPN category includes diseases with both myelodysplastic and myeloproliferative features. The revised International Consensus Classification now requires the presence of increased peripheral blood cell counts and concomitant cytopenia for diagnosis. The use of modern gene sequencing has improved understanding of the biological characteristics and specific mutations of these diseases, which are now included in the diagnostic criteria for some MDS/MPN entities. This review highlights the changes in diagnostic criteria and provides practical guidance for diagnosis.
Review
Hematology
Sa A. Wang, Attilio Orazi, Jason Gotlib, Andreas Reiter, Alexandar Tzankov, Robert P. Hasserjian, Daniel A. Arber, Ayalew Tefferi
Summary: Based on new data and increased understanding of disease molecular genetics, the international consensus classification has made changes in the diagnosis and classification of eosinophilic disorders and systemic mastocytosis. The changes include renaming myeloid/lymphoid neoplasms with eosinophilia and gene rearrangements, expanding the category to include more gene fusions, and introducing bone marrow morphologic criteria. The review focuses on the updates related to these diseases, including changes in morphology, molecular genetics, clinical features, prognosis, and treatment.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Oncology
Craig W. Freyer, Mitchell E. Hughes, Alison Carulli, Adam Bagg, Elizabeth Hexner
Summary: MLNFGFR1 is a rare and aggressive neoplasm with poor prognosis. Pemigatinib has shown high response rates and represents a significant advance in the management of MLNFGFR1. Long-term efficacy and the use of pemigatinib in combination with chemotherapy and after alloHSCT need further investigation.
EXPERT REVIEW OF ANTICANCER THERAPY
(2023)
Article
Hematology
A. Samer Al-Homsi, Frank Cirrone, Stephanie Wo, Kelli Cole, Andres Suarez-Londono, Sharon L. Gardner, Jingmei Hsu, Kelsey Stocker, Benedetto Bruno, Judith D. Goldberg, Benjamin A. Levinson, Maher Abdul-Hay
Summary: The combination of PTCy, abatacept, and a short course of tacrolimus (CAST) after haploidentical HSCT is safe and effective in reducing the incidence of grades 2-4 acute GVHD. The study enrolled 46 patients with a median age of 60 years, and the cumulative incidences of grades 2-4 and 3 or 4 acute GVHD were 17.4% and 4.4%, respectively. The CAST regimen also showed favorable outcomes in terms of treatment-related mortality, chronic GVHD rate, relapse rate, progression-free survival, and overall survival.
Article
Hematology
Yu Akahoshi, Nikolaos Spyrou, William J. Hogan, Francis Ayuk, Zachariah DeFilipp, Daniela Weber, Hannah K. Choe, Elizabeth O. Hexner, Wolf Roesler, Aaron M. Etra, Karamjeet Sandhu, Gregory A. Yanik, Chantiya Chanswangphuwana, Carrie L. Kitko, Ran Reshef, Sabrina Kraus, Matthias Woelfl, Matthias Eder, Hannah Bertrand, Muna Qayed, Pietro Merli, Stephan A. Grupp, Paibel Aguayo-Hiraldo, Tal Schechter, Evelyn Ullrich, Janna Baez, Rahnuma Beheshti, Sigrun Gleich, Steven Kowalyk, George Morales, Rachel Young, Deukwoo Kwon, Ryotaro Nakamura, John E. Levine, James L. M. Ferrara, Yi-Bin Chen
Summary: This study aims to better describe the clinical progression and prognosis of late acute graft-versus-host disease (GVHD). Late acute GVHD was more severe than classic acute GVHD based on clinical and biomarker parameters, and had a lower overall response rate after treatment. Advanced age, female-to-male sex mismatch, and the use of reduced intensity conditioning were associated with the development of late acute GVHD. Overall outcomes were comparable between patients with classic and late acute GVHD, suggesting that treatment strategies based on clinical presentation are appropriate.
Article
Hematology
Ayalew Tefferi, Maymona Abdelmagid, Aref Al-Kali, Mrinal Patnaik, William J. Hogan, Kebede Begna, Naseema Gangat, Attilio Orazi, Dong Chen, Kaaren K. Reichard, Animesh Pardanani
Summary: This study demonstrates the independent prognostic contribution of the ICC system for SM-Adv and the Mayo alliance risk factors for survival in SM.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Hematology
Monzr M. Al Malki, Kaitlyn London, Janna Baez, Yu Akahoshi, William J. Hogan, Aaron Etra, Hannah Choe, Elizabeth Hexner, Amelia Langston, Sunil Abhyankar, Doris M. Ponce, Zachariah Defilipp, Carrie L. Kitko, Kehinde Adekola, Ran Reshef, Francis Ayuk, Alexandra Capellini, Chantiya Chanswangphuwana, Matthias Eder, Gilbert Eng, Isha Gandhi, Stephan Grupp, Sigrun Gleich, Ernst Holler, Nora Rebeka Javorniczky, Stelios Kasikis, Steven Kowalyk, George Morales, Umut Oezbek, Wolf Roesler, Nikolaos Spyrou, Gregory Yanik, Rachel Young, Yi-Bin Chen, Ryotaro Nakamura, James L. M. Ferrara, John E. Levine
Summary: The combination of natalizumab and corticosteroids did not improve outcomes for patients with newly diagnosed high-risk GVHD.
Article
Pediatrics
Alexander Maad El-Ali, Selin Ocal, C. Austen Hartwell, Judith D. D. Goldberg, Xiaochun Li, Jaimelee Prestano, Ranjith Kamity, Laura Martin, Naomi Strubel, Shailee Lala
Summary: This retrospective study evaluated predictive factors for diagnostic ultrasound (US) for midgut volvulus and found that US was most frequently diagnostic in patients with bilious emesis or age less than 28 days. A non-diagnostic US for midgut volvulus requires a predetermined follow-up strategy to investigate potential midgut volvulus.
PEDIATRIC RADIOLOGY
(2023)
Article
Hematology
Matthew P. Connor, Alison W. Loren, Elizabeth O. Hexner, Mary Ellen Martin, Saar I. Gill, Selina M. Luger, James K. Mangan, Alexander E. Perl, Shannon R. McCurdy, Keith W. Pratz, Colleen Timlin, Craig W. Freyer, Alison Carulli, Christopher Catania, Jacqueline Smith, Lauren Hollander, Alexis M. Zebrowski, Edward A. Stadtmauer, David L. Porter, Noelle Frey
Summary: Patients with refractory or relapsed and refractory myeloid malignancies have a poor prognosis. The use of a myeloablative conditioning regimen combining clofarabine with busulfan has shown antileukemic activity with acceptable toxicity in patients under 70 years old. This study evaluates the clinical outcomes of patients with active myeloid malignancies undergoing allogeneic hematopoietic cell transplantation (HCT) with the clofarabine-busulfan regimen, and concludes that it is an effective transplantation strategy, particularly for patients with advanced myelodysplastic syndrome (MDS).
TRANSPLANTATION AND CELLULAR THERAPY
(2023)