Cold Agglutinin Disease: Treatment Goals

Clinicians offer their perspectives on establishing treatment goals for patients with cold agglutinin disease and describe how they measure treatment response.

Neil Minkoff, MD: The question that comes to mind is—you’ve identified this, before we even get into the options for treatment—what’s your goal for your patient?

Mihir Raval, MD, MPH: The goals of the treatment I would divide mainly into symptom management, which means we’re looking at correction of underlying anemia that’s predominantly causing that fatigue, tiredness, weakness, or decline in their activities of daily living. That’s the No. 1 treatment. No. 2 is cold-induced symptom management. That means if the patients are having symptoms of extremities, or just as Dr Lorber mentioned about the cold ear and nose tips, those are some of the symptoms that are limiting patients’ activities of daily living, like hanging out with their families and going out to eat during winter months. Those are 2 predominant symptoms that you can manage. The third goal is actually preprocedural correction. Sometimes, patients may have cold agglutinin disease that has to undergo a surgery. That may warrant for some urgent intervention, such as plasma exchange. The fourth treatment goal is long-term management to tackle the underlying problem, the clone that may be causing this problem by looking at either B-cell–directed treatment or by doing some complement-directed treatment, depending on how severe the disease is.

Neil Minkoff, MD: How do you measure the patient’s response? Is it mostly how they feel? Is it a combination of that with lab data? When you decide that you’ve accomplished your goal? Is that a silly question?

Mihir Raval, MD, MPH: No, it’s a very valid question. But as a clinician, when I decide that this patient needs the treatment, my assessment to treatment response is multifocal, so it’s not just 1 area that I’m looking at. I’m looking at lab improvement. Sometimes, those patients’ hemolysis doesn’t completely get better but is good enough that they can have a better quality of life and can be followed periodically to make sure that it’s not worsening. That’s 1 goal: assessing the lab improvement, their hemoglobin improved enough that they are transfusion independent. That’s 1 of the biggest goals. The second goal is, do their symptoms improve enough that they can live a little better life without having cold-related symptoms? Improvement in the fatigue and also simultaneously monitoring for any concerns of progressive lymphoma or autoimmune disease. Those are some of the monitors that I follow to assess the treatment response.

Transcript edited for clarity.

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