Table 4.
Symptom improvement: MPN-SAD and PGIC.
| Time point, % improvement, cumulative* | Lead-in cohort (N = 21) | |||||
|---|---|---|---|---|---|---|
| MPN-SAD†n (%) |
PGIC improvement **n (%) |
|||||
| TSS | Spleen‡ | Constitutional§ | nTSSǁ | |||
| Monthly mean at week 12 | Cycle 4 day 1 | |||||
| ≥50% | 2 (9.52) | 6 (28.57) | 3 (14.29) | 4 (19.05) | Very much | 1 (4.76) |
| ≥30% | 5 (23.81) | 7 (33.33) | 4 (19.05) | 6 (28.57) | Much | 0 |
| ≥20% | 8 (38.10) | 8 (38.10) | 6 (28.57) | 7 (33.33) | Minimally | 4 (19.05) |
| Monthly mean at week 24 | Cycle 7 day 1 | |||||
| ≥50% | 1 (4.76) | 2(9.52) | 2(9.52) | 1 (4.76) | Very much | 0 |
| ≥30% | 3 (14.29) | 4 (19.05) | 3 (14.29) | 3 (14.20) | Much | 1 (4.76) |
| ≥20% | 4 (19.05) | 5 (23.81) | 3 (14.29) | 4 (19.05) | Minimally | 2 (9.52) |
| Severe Subset†† (n = 14) | ||||||
| Monthly mean at week 12 | Cycle 4 day 1 | |||||
| ≥50% | 2 (14.29) | 4 (28.57) | 2 (14.29) | 2 (14.29) | Very much | 1 (7.14) |
| ≥30% | 4 (28.57) | 5 (35.71) | 3 (21.43) | 4 (28.57) | Much | 0 |
| ≥20% | 6 (42.86) | 6 (42.86) | 5 (35.71) | 5 (35.71) | Minimally | 2 (14.29) |
| Monthly mean at week 24 | Cycle 7 day 1 | |||||
| ≥50% | 1 (7.14) | 2 (14.29) | 0 | 1 (7.14) | Very much | 0 |
| ≥30% | 1 (7.14) | 3 (21.43) | 1 (7.14) | 1 (7.14) | Much | 1 (7.14) |
| ≥20% | 2 (14.29) | 3 (21.43) | 1 (7.14) | 2 (14.29) | Minimally | 1 (7.14) |
MPN-SAD = Myeloproliferative Neoplasm Symptom Assessment Diary; nTSS = new total symptom score; PGIC = Patient Global Impression of Change.
The cumulative frequency of patients is the total of each frequency added to its predecessor. The cumulative percentage of patients divides the cumulative frequency by the total number of observations.
Higher scores are associated with better health for functional scales and worse health for symptom scales.
Spleen-Related Symptom Score is the sum of early satiety and abdominal discomfort.
Constitutional Symptom Score is the sum of fatigue, night sweats, pruritus, and bone pain.
nTSS is the sum of spleen-related symptoms and constitutional symptoms.
Since starting the study medication, my myelofibrosis symptoms are: 1 = Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = No change; 5 = Minimally worse; 6 = Much worse; 7 = Very much worse.
Severe Subset are patients with a baseline (7-day average) MPN-SAD severity score ≥5 (on 0–10 scale) on at least one, or a severity score ≥3 on at least two of the following items: fatigue, early satiety, abdominal discomfort, inactivity, night sweats, pruritus, and/or bone pain.