eCase - Hematology 2022: Nonmalignant diseases

Key Studies in Nonmalignant Hematology

At the 2022 American Society of Hematology (ASH) Annual Meeting, new clinical trial data in nonmalignant hematologic disorders were reported, including novel therapies for cold agglutinin disease (CAD), paroxysmal nocturnal hemoglobinuria (PNH), graft-vs-host disease (GVHD), immune thrombocytopenia (ITP), and hemophilia.

In this module, Catherine M. Broome, MD; David Dingli, MD, PhD; Corey Cutler, MD, MPH, FRCPC; Marshall Mazepa, MD; and Allison Wheeler, MD, MSCI, review and discuss the clinical implications of key studies in these hematologic disorders that were presented at ASH 2022.

Please note that the slide thumbnails in this activity link to brief PowerPoint slidesets that also can be found here, each focused on the specific study or topic of interest. These slidesets also may be downloaded by clicking on any of the thumbnails within the activity.

Clinical Care Options plans to measure the educational impact of this activity. A few questions will be asked twice: Once at the beginning of the activity and then once again after the discussion that informs the best choice. Your responses will be aggregated for analysis, and your specific responses will not be shared.

Before continuing with this educational activity, please take a moment to answer the following questions.

Your patient with CAD mentions that she is experiencing fatigue and has had low hemoglobin (≤10 g/dL) in her previous 2 laboratory assessments. At ASH 2022, data were presented from the phase III CADENZA study of sutimlimab, a selective classical complement pathway inhibitor, vs placebo in patients with CAD and no recent history of transfusion. The study included analyses evaluating the role of sutimlimab on patients’ fatigue and other quality of life measures, with the goal of expanding treatment options for patients with the disease. As part of your recommendation to try out this new treatment, you would mention to her that all of these results were reported for the CADENZA study EXCEPT which one?

A.
A significant increase in hemoglobin and decrease in bilirubin by Week 26
B.
Improvement in FACIT-Fatigue in patients with sutimlimab, including those switching from placebo at Week 26
C.
Improvement in laboratory values and FACIT-Fatigue scores that were not sustained through Week 86
D.
Improvement reported in physical and mental component scores at last on-treatment visit on Week 87

At ASH 2022, De Latour and colleagues reported results from the phase III APPLY-PNH study of iptacopan vs standard of care of eculizumab or ravulizumab in patients with PNH and residual anemia while receiving standard therapy. The study was designed to establish superiority for iptacopan vs standard of care complement 5 inhibition (ravulizumab vs eculizumab). Which of the following most accurately reflects the results reported for this study regarding major adverse vascular events?

A.
Both intravascular and extravascular hemolysis improved
B.
Hemoglobin increased, but intravascular hemolysis was unchanged
C.
Intravascular and extravascular hemolysis remained unchanged
D.
Intravascular hemolysis improved, but extravascular hemolysis was unchanged

Which patient population has an unmet need and could benefit MOST from enrollment on a phase III fitusiran clinical trial?

A.
Hemophilia A without inhibitors
B.
Hemophilia A with inhibitors
C.
Hemophilia B without inhibitors
D.
Hemophilia B with inhibitors
E.
Hemophilia A or B without inhibitors

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Key Studies in Nonmalignant Hematology: Independent Conference Coverage of ASH 2022

Catherine M. Broome, MD

Corey Cutler, MD, MPH, FRCPC

David Dingli, MD, PhD

Marshall Mazepa, MD

Allison P Wheeler, MD, MSCI

Activity

Key Studies in Nonmalignant Hematology

Which patient population has an unmet need and could benefit MOST from enrollment on a phase III fitusiran clinical trial?