Associate Professor, Jane Anne Nohl Division of Hematology, Keck School of Medicine, University of Southern California

Caroline Piatek, MD

Piatek discussed findings from a healthcare resource utilization study and a sentiment analysis study presented at ASH 2024.

Caroline Piatek, MD: High HCRU, Patient Concerns Highlight Great Unmet Need in wAIHA

New research has revealed high unmet needs in people with warm autoimmune hemolytic anemia (wAIHA), as evidenced by high healthcare resource utilization (HCRU) and prevalent concerns about adverse events (AEs) and a lack of treatment efficacy.

These findings are from 2 studies, data from which were presented at the

 66th American Society of Hematology (ASH) Annual Meeting and Exposition

, held December 7-10, 2024, in San Diego, California.

® spoke with Caroline Piatek, MD, Associate Professor, Jane Anne Nohl Division of Hematology, Keck School of Medicine, University of Southern California, who served as an investigator on both studies.

In a retrospective, HCRU study, Piatek and colleagues found that HCRU remained high in both 12-month periods preceding and following initial wAIHA diagnosis in people with both primary and secondary wAIHA.

 In a sentiment analysis study powered by artificial intelligence (AI), they found a dearth of positive sentiments about rituximab treatment or transfusion therapy, notable concerns about AEs and a lack of treatment efficacy, and a prevalence of information seeking, highlighting the importance of healthcare provider support and involvement in implementing a successful treatment strategy.

During the discussion, Piatek emphasized the high unmet need that these studies revealed and the need for better treatment strategies for people with wAIHA.

“There just isn't much [out there for wAIHA]. And so, anything is helpful, but ultimately, we need better therapeutic agents in this setting… Characterizing the disease burden is important, but we kind of have to move forward from that and do something about it,” Piatek said.

Jackson LA, Pesa J, Choudhry Z, et al. A Retrospective Database Analysis of Healthcare Resource Utilization in Patients with Warm Autoimmune Hemolytic Anemia in the United States. Presented at: ASH Annual meeting; December 7-10; San Diego, California. Abstract 2324

Piatek C, Jackson LA, Brethenoux C, et al. Sentiment Analysis Applied to Digital Conversations Among Warm Autoimmune Hemolytic Anemia (wAIHA) Patients Receiving Rituximab and/or Blood Transfusion. Presented at: ASH Annual meeting; December 7-10; San Diego, California. Abstract 3705

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Piatek shared the latest data update from the phase 3 ALPHA study at the ASH 2024.

Caroline Piatek, MD: Improving Patient-Reported Outcomes in PNH With Danicopan Add-on Therapy

Long-term danicopan add-on therapy to ravulizumab or eculizumab in people with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (csEVH) resulted in sustained improvements in patient-reported fatigue, QoL and physical function.

These findings are from long-term follow-up data for up to 72 weeks in the phase 3 ALPHA study and were presented at the

, held December 7-10, 2024, in San Diego, California, by Caroline Piatek, MD, Associate Professor, Jane Anne Nohl Division of Hematology, Keck School of Medicine, University of Southern California.

“One of the issues with PNH is that patients report a lot of fatigue, and so having medications that can improve that symptom for the patients is clinically meaningful,” Piatek told 

The ALPHA study is a double-blind, placebo-controlled clinical trial that randomized adult patients with PNH, csEVH, and at least 6 months’ treatment with ravulizumab or eculizumab to danicopan (n = 57) or placebo (n = 29). Of all participants, 54 and 26, respectively, completed the study up to week 24; and 46 and 24 patients, respectively, completed the long-term extension.

Piatek and colleagues found that at week 12, change from baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) score was significantly greater for patients receiving danicopan compared with those receiving placebo in the double-blinded phase (least-squares mean, 8.13 [95% CI, 6.30-9.96] vs 2.53 [95% CI, −0.22-4.91], respectively; 

= .0004). European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC-QLQ-C30) fatigue subscale scores in the danicopan arm improved from a mean 43.3 (standard deviation [SD], 27.2) at baseline to 27.4 (SD, 20.3), 25.4 (SD, 23.6), and 27.8 (SD, 22.2) at weeks 12, 24, and 72, comparable with the general population norm (29.5); whereas in the placebo–danicopan arm scores improved from 49.0 (SD, 25.8) at baseline to 47.1 (SD, 28.6), 37.5 (SD. 27.8), and 44.9 (SD, 24.2), respectively.Mean EQ-5D-3L scores largely remained stable.

Piatek C, Lee JW, Griffin N, et al. Danicopan As Add-on Therapy to Ravulizumab or Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Patient-Reported Outcomes from the Phase 3 ALPHA Trial. Presented at: Presented at: ASH Annual meeting; December 7-10; San Diego, California. Abstract 2692.

Caroline Piatek, MD

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