Qualitative Examination of Treatment Experiences Among Individuals Living with Warm Autoimmune Hemolytic Anemia (wAIHA)

Purpose: The purpose of this qualitative focus group was to gain an understanding of the treatment journey of people living with wAIHA. Treatment experience, burden, and level of satisfaction particularly with oral corticosteroids (OCS), rituximab, and splenectomy were examined. Patient management of treatment side effects and adverse events was also explored.

Background: wAIHA is a rare, chronic relapsing autoimmune disorder characterized by the destruction of red blood cells (Brodsky RA. N Engl J Med 2019). wAIHA is associated with a significant physical and emotional burden on patients and is shown to impact health related quality of life (HRQoL) (Hill QA. Br J Haematol 2021).

Methods: US members of a patient council participated in a 2-hour virtual focus group to explore the treatment journey experienced by wAIHA patients. Participants included 5 individuals ranging in age from their 30's to 70's. Four of the participants were female. One participant identified as African American and 4 identified as white. A structured moderator guide directed the discussion which was transcribed and reviewed for themes by 2 reviewers.

Results: The themes identified for participant treatment experiences included: management of side effects and the impact of fatigue on participant's lives. All participants experienced adverse events/side effects from at least one medication (primarily OCS). Three females cited mood swings/emotional impact and hot flashes/frequent sweating. All participants had been prescribed OCS at least once for the treatment of wAIHA, for varying lengths of times. On higher doses of OCS (prednisone 60mg-100mg), 3 participants expressed how quickly and severely they would experience side effects (e.g., nausea, sweating, swollen ankles/legs) with a missed dose. Overall, the group had mixed levels of satisfaction with OCS due to the balance between the side effects and how well and how quickly it improved symptoms when experiencing a relapse. Three participants were not currently on OCS and shared experiences related to the length of time it took to taper off the treatment, and the desire to avoid future OCS use due to side effects. Four of the participants had received rituximab, experiencing few side effects (muscle spasms, sleeplessness, and shaking) but having mixed levels of satisfaction due to time commitment and burden (e.g. long infusion times, pre-medication grogginess) versus the level of effectiveness.

Other important treatment-related themes were observed. Two participants had undergone a splenectomy, and neither reported a notable benefit from this surgery, questioning their decision after the fact. Four participants had not experienced fatigue prior to their diagnosis of wAIHA. Once diagnosed with wAIHA, fatigue became a large burden to their everyday lives. All 5 participants had to modify their working arrangements because of wAIHA diagnosis and/or symptoms. Participants described the emotional toll of attempting to convey the intensity of their fatigue to their loved ones and health care providers. Four participants stated they were provided with inadequate information about what to expect for the medication side effects, including blood clots. All desired more information and education from their health care providers about potential side effects and ways to mitigate/prevent them.

Conclusion: Participants described a variety of wAIHA treatment-related experiences, burdens, and levels of satisfaction with current treatment options. wAIHA treatment-related experience reported by participants included side effects with OCS, the burdens associated with receiving rituximab, the lack of benefit of splenectomy, the toll of fatigue, and a general lack of knowledge on how to manage treatment side effects. Although limited by the small sample size, these results indicate that newer, more effective wAIHA treatment options with improved side effect and efficacy profiles are needed to better address the unmet needs of this patient population.

Piatek:Apellis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Rigel: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees; Alpine Immune Science: Research Funding; Zenas BioPharma: Research Funding; Novartis: Membership on an entity's Board of Directors or advisory committees; Argenx: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Alexion, AstraZeneca Rare Disease: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Sanofi: Membership on an entity's Board of Directors or advisory committees; Sobi: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Omeros: Membership on an entity's Board of Directors or advisory committees. Rodriguez:Janssen Scientific Affairs, LLC: Current Employment; Johnson & Johnson: Current equity holder in publicly-traded company. Choudhry:Janssen Scientific Affairs, LLC: Current Employment; Johnson & Johnson: Current equity holder in publicly-traded company; Takeda: Current equity holder in publicly-traded company. Jackson:Janssen Scientific Affairs, LLC: Current Employment; Johnson & Johnson: Current equity holder in publicly-traded company. Shea:Janssen Scientific Affairs, LLC: Current Employment; Johnson & Johnson: Current equity holder in publicly-traded company. Sandorfi:Johnson and Johnson: Consultancy; BMS: Consultancy.