QoL outcomes for patients treated with HU or IFNα

Myeloproliferative neoplasms (MPN) are widely associated with a high-disease-related symptom burden and lower quality of life (QoL) compared with individuals without the disease.¹ Currently, there are limited data on the relation between QoL and the type of cytoreductive therapy in patients with MPN.¹

In response to this, during the 64th American Society of Hematology (ASH) Annual Meeting and Exposition, Knudsen¹ presented results from the phase III DALIAH trial (NCT01387763) investigating QoL and symptom burden of patients with MPN treated with either hydroxyurea (HU) or pegylated interferon alpha-2 (IFNα-2). The DALIAH study design has previously been reported on the MPN Hub and we are pleased to summarize the results below.

Results

Baseline symptom burden

The mean total symptom score (TSS), defined as a score range of 0–100 (with 100 representing the highest level of symptom severity), at baseline for all patients was 14.8.¹ The mean TSS at baseline for each MPN subtype is shown in Table 1. Clinical deficient overall QoL (score ≥4 on the MPN symptom assessment form survey) was reported in 41% of patients. At baseline, fatigue was the most common symptom (82% of patients) and had the highest mean symptom intensity (Figure 1).¹

Table 1. Mean TSS at baseline for each MPN subtype*

Figure 1. Mean symptom severity at baseline* 

*Adapted from Knudsen.^1
†Mean severity defined as the average score out of 10 from the MPN symptom assessment survey form for each individual symptom.

One or more moderate or severe symptoms were reported by 70% of patients, with the most common including²:

• Fatigue (25%)
• Concentration problems (21%)
• Insomnia (20%)

Severe symptoms were reported in 46% of patients.² Overall, baseline characteristics, TSS, and individual symptoms were balanced between the two treatment groups, except abdominal pain which was higher in patients treated with IFNα2a (p = 0.02).²

Symptom burden during treatment²

A total of 179 patients completed at least one MPN symptom assessment form survey, while 96 patients completed a survey at the 36-month time point. The overall rate of treatment discontinuation was 48%. During HU treatment, patients experienced both temporary and lasting worsening of individual symptoms (p < 0.05), including:

• Fatigue
• Abdominal pain
• Abdominal discomfort
• Sexuality problems
• Itching
• Bone pain

Patients treated with IFNα also experienced declines in individual symptoms (p < 0.05), including:

• Fatigue
• Early satiety
• Inactivity
• Headache
• Concentration problems
• Insomnia

Overall, TSS increased within the first year for all patient groups; however, it was significantly higher in patients treated with HU at 36 months compared with baseline (p = 0.02). At the 36-month time point, the percentage of patients who experienced a reduction in TSS was comparable between the treatment groups (Figure 2).

Figure 2. Patients who experienced a TSS reduction at 36 months* 

HU, hydroxyurea; IFNα, interferon alpha; TSS, total symptom score.
*Adapted from Knudsen, et al.²

At 36 months, ≥1 moderate or severe symptom was experienced by 63% of patients. Fatigue remained the most common symptom across all patient groups and there was no significant difference in individual symptom scores or proportion of moderate or severe symptoms reported between patient groups.

Conclusion

Results from the study show TSS increased in patients who were treated with either HU or IFNα and that there was no significant difference in TSS between treatment groups. At the 30- and 36-month time points, patients treated with HU had significantly increased TSS compared with baseline. Overall, patients with a high baseline TSS experienced the greatest improvement in TSS during the course of treatment with either HU or IFNα.