Quality of life after immune suppressive therapy in aplastic anemia

TL;DR

This study examines how quality of life is affected in aplastic anemia patients after immune suppressive therapy, finding ongoing physical and psychological challenges.

Contribution

The study introduces the use of a newly developed AA-specific quality of life questionnaire to assess long-term outcomes after IST.

Findings

Fatigue was reported by 83% of patients despite successful IST.

Patients with partial response showed a trend toward better psychological wellbeing compared to those with complete response.

Quality of life scores showed no significant difference between complete and partial responders, but psychological domains showed notable trends.

Abstract

Acquired aplastic anemia (AA) is a rare form of immune-mediated bone marrow failure, which can result in life-threatening infections or bleeding if left untreated. Treatment consists of either immune suppressive therapy (IST) or allogeneic stem cell transplantation (alloHSCT). While considerable research has been published regarding survival, response rate and toxicity of both treatments, knowledge on the impact on quality of life (QoL) is scarce. We used the recently developed AA-specific QoL questionnaire (QLQ-AA/PNH-54) to evaluate QoL in a single center cohort of AA patients who were successfully treated with IST. The 54 questions represent 12 different QoL domains. Results were analyzed for all patients and grouped based on hematologic response (complete response (CR) or partial response (PR)). Thirty-six successfully treated adult patients (15 in CR, 21 in PR) completed the questionnaire (median age 54 years, range 21–71; median time since last IST 5 years, range 0–41). Fatigue was experienced by 83% of patients. Even though total QoL scores did not significantly differ between patients with PR and CR (105 vs 92, p-value 0,17) there appeared to be a trend towards higher scores in patients with PR, especially in domains concerning psychological wellbeing. This trend was most clear in the domains fear of progression (2,12 in PR patients vs 1,73 in CR patients; p-value 0,08) and role functioning (2,22 vs 1,88; p-value 0,07). In conclusion, patients with AA continue to experience psychological and physical effects despite successful IST.

The online version contains supplementary material available at 10.1007/s00277-024-05731-x.

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