JTH in clinic: A new series for the practicing clinician

Abstract

While the practice of medicine today involves substantially more knowledge of the science underpinning disease than was available decades ago, the need to include the “art” in the practice of clinical medicine remains. New discoveries in basic science are now more rapidly translated into clinical drug development and evolution in understanding of the pathophysiology of disease, which can lead seemingly overnight to new diagnostic approaches and paradigm shifts in treatments. While randomized controlled clinical trials (RCTs) are considered the gold standard for studying the safety and efficacy of a treatment, with the goal of reducing bias and examining the cause–effect relationships between an intervention and outcome, many disorders in hemostasis and thrombosis do not lend themselves to randomized controlled trials, either due to low prevalence in the population or high‐risk patient populations making such studies difficult to conduct. Clinicians may also be faced with a patient that would not have been eligible for the RCT yet requires treatment; guidance to understand how to apply the available data to their individual patient is required. With the January 2021 Journal of Thrombosis and Haemostasis (JTH) volume, we are launching an innovative article series titled “JTH in Clinic” to be published every 2 months, to address this discordance between scientific knowledge and clinical care. The JTH in Clinic articles will bridge the gap between knowledge and practice by addressing a specific clinical problem in thrombosis and hemostasis. Using a case‐based format, senior experienced clinicians will lead the reader through the care of the patient from identifying the signs and symptoms at presentation, to diagnosis and treatment, highlighting data that are known, as well as areas of controversy and uncertainty. Expert opinion on the management of these often‐complex cases will be provided to give the practicing clinician the guidance needed when faced with the individual patient with a disorder of thrombosis or hemostasis. JTH in Clinic articles are not intended to be an exhaustive review of the literature but will capture current thinking and summarize the state‐of‐the art diagnostic and therapeutic modalities for the clinician in a succinct and informative article. Articles will include topics on the clinical management of patients as well approaches to using diagnostic and research testing for disorders of thrombosis and hemostasis. Our first manuscript in the JTH in Clinic series, by Pico, Veyradier, and Coppo, examines the question “Should all patients with immune‐mediated thrombotic thrombocytopenic purpura (iTTP) receive caplacizumab?” Caplacizumab, an anti‐von Willebrand factor (VWF) nanobody that blocks the interaction between platelets and VWF to prevent platelet aggregation and microthrombus formation, has recently been approved for use in Europe, Canada, and the United States. Given the rarity of the occurrence of iTTP, however, many practitioners have no direct knowledge of how to use this newly available agent in clinical practice. Drawing from randomized clinical trial data and published real‐world experience from their own and other centers, the authors present a pragmatic case‐based approach to determining when to initiate caplacizumab and when to discontinue, as well as contra‐indications for use. Pico, Veyradier, and Coppo also discuss the cost of caplacizumab in the context of iTTP care, a concern for many clinicians and hospital formularies. We hope that this series will address the needs of the readers of JTH and we look forward to your comments. The author has no conflicts of interest to report. Jean M. Connors conceived and wrote the manuscript.