Developing the evidence base for the management of autoimmune haemolytic anaemia (AIHA): the UK experience

Hill, Quentin A.; Horan, Matthew; Charlton, Adrian J.; Bullock, Tom; Massey, Edwin; Hill, Anita; Allard, Shubha; Stanworth, Simon

doi:10.1111/bjh.17217

Cited by 5 publications

(4 citation statements)

References 11 publications

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“…The negative impact of CAD‐associated symptoms on participants' lives ranged from minimal (e.g., wearing extra clothing) to substantial (e.g., stop working, change in retirement plans). There is limited awareness of CAD in the medical community, and a lot is still unknown in terms of clinical features and outcomes 3,4,22 . Recent data indicate that, while some cold‐induced symptoms may be transient, patients with CAD can experience persistent chronic hemolysis regardless of season 23 .…”

Section: Discussionmentioning

confidence: 99%

“…There is limited awareness of CAD in the medical community, and a lot is still unknown in terms of clinical features and outcomes. 3,4,22 Recent data indicate that, while some cold-induced symptoms may be transient, patients with CAD can experience persistent chronic hemolysis regardless of season. 23 The insights from our interviews with adults with CAD highlight a need to better understand and quantify the impact of CAD and its treatment on patient quality of life.…”

Section: Elimination or Minimization Of Reactions To Cold Environmentsmentioning

confidence: 99%

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Development of the Cold Agglutinin Disease Symptoms and Impact Questionnaire (CAD‐SIQ)

Joly

DasMahapatra

DiBenedetti

et al. 2023

European J of Haematology

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Objectives: Cold agglutinin disease (CAD) is a rare autoimmune hemolytic anemia. This study aimed to identify disease-related symptoms and impacts important to patients with CAD, and to develop a novel CAD-specific patient-reported outcome measure.Methods: Adults with CAD were randomly selected from a United States patient panel to participate in concept elicitation (CE) interviews to identify important symptoms and impacts or cognitive debriefing (CD) interviews to assess the comprehension and relevance of the draft item set.Results: Overall, 37 adults were included (mean [range] age 67.2 [35-87] years). In CE interviews (n = 16), the most frequently reported CAD-related symptoms were reactions to cold environments and fatigue (both 93.8%). CAD had negative impacts on enjoyable activities (81.3%) and daily activities (75.0%). Following CE, standard survey methodological principles were used to develop a draft item pool of 14 concepts. Items were refined through three iterative rounds of CD interviews (n = 21), yielding 11 final items: fatigue; cold sensitivity; dyspnea; wearing extra clothing; limited physical, social, and enjoyable activities; difficulty with usual activities; mood; frustration; and anxiety/stress. Conclusions:The novel 11-item CAD-Symptoms and Impact Questionnaire provides a measure of the symptoms and impacts of CAD that are important to patients. K E Y W O R D S cold agglutinin disease, conceptual model, instrument development, patient interviews, patientreported outcome Novelty statements What is the new aspect of your work?This work describes the development of a novel patient-reported outcome (PRO) measure, the CAD-Symptoms and Impact Questionnaire (CAD-SIQ), which, to our knowledge, is the first PRO measure designed to assess the patient experience with CAD.

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Section: Discussionmentioning

confidence: 99%

Section: Elimination or Minimization Of Reactions To Cold Environmentsmentioning

confidence: 99%

Development of the Cold Agglutinin Disease Symptoms and Impact Questionnaire (CAD‐SIQ)

Joly

DasMahapatra

DiBenedetti

et al. 2023

European J of Haematology

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“…Such a therapy does currently not exist. With this in mind, more prospective clinical trials are needed ( 12 , 14 , 15 ). Patients requiring therapy in any line may be considered for a prospective trial if eligible, provided randomization to the control arm (if relevant) is considered ethically justifiable.…”

Section: How To Select Therapy In Warm Aihamentioning

confidence: 99%

“…It was not until 1938 that Dameshek & Schwartz draw a distinction between congenital and acquired hemolytic anemia and, two years later, postulated an immune mechanism based on the finding of hemolysin in patient sera ( 11 ). Until the turn of the millennium, treatment was mostly based on theoretical considerations, clinical experience, and expert opinion, but during the last two decades, several prospective studies have been conducted ( 1 , 12 – 14 ). Currently, several new therapies have appeared and quite a few clinical trials are running ( 15 , 16 ).…”

Section: Introductionmentioning

confidence: 99%

The choice of new treatments in autoimmune hemolytic anemia: how to pick from the basket?

2023

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Autoimmune hemolytic anemia (AIHA) is defined by increased erythrocyte turnover mediated by autoimmune mechanisms. While corticosteroids remain first-line therapy in most cases of warm-antibody AIHA, cold agglutinin disease is treated by targeting the underlying clonal B-cell proliferation or the classical complement activation pathway. Several new established or investigational drugs and treatment regimens have appeared during the last 1-2 decades, resulting in an improvement of therapy options but also raising challenges on how to select the best treatment in individual patients. In severe warm-antibody AIHA, there is evidence for the upfront addition of rituximab to prednisolone in the first line. Novel agents targeting B-cells, extravascular hemolysis, or removing IgG will offer further options in the acute and relapsed/refractory settings. In cold agglutinin disease, the development of complement inhibitors and B-cell targeting agents makes it possible to individualize therapy, based on the disease profile and patient characteristics. For most AIHAs, the optimal treatment remains to be found, and there is still a need for more evidence-based therapies. Therefore, prospective clinical trials should be encouraged.

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Real-world data from India on clinical practices in the management of autoimmune haemolytic anaemia: A survey-based cross-sectional assessment

Das¹,

Das²,

Shastry³

et al. 2023

Transfusion Clinique et Biologique

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Developing the evidence base for the management of autoimmune haemolytic anaemia (AIHA): the UK experience

Cited by 5 publications

References 11 publications

Development of the Cold Agglutinin Disease Symptoms and Impact Questionnaire (CAD‐SIQ)

Development of the Cold Agglutinin Disease Symptoms and Impact Questionnaire (CAD‐SIQ)

The choice of new treatments in autoimmune hemolytic anemia: how to pick from the basket?

Real-world data from India on clinical practices in the management of autoimmune haemolytic anaemia: A survey-based cross-sectional assessment

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