Page 88 - 2020_09-Haematologica-web

A.L. Sørensen et al.
years was 12% (95% CI: 6-22%) compared with 47% (95% CI: 33-61%) at baseline. In 33 of 44 JAK2 V617F- mutated patients, measurements of the JAK2 V617F allele burden were available from within the year before inclu- sion. There were no significant differences in the JAK2 V617F allele burden before inclusion compared with base- line. Of the 44 patients with the JAK2 V617F mutation, one (2%) achieved CMR, and 17 (39%) achieved PMR within 2 years of treatment (Figure 2B). Setting the limit of CMR at 1%, four (9%) patients achieved CMR, and 16 (36%) achieved PMR within 2 years of treatment (Online Supplementary Figure S3). Further analyses were done with a limit of CMR 1-5% (Online Supplementary Table S1). We observed no difference in the JAK2 V617F allele burden change between patients with PV and patients with MF (Online Supplementary Figure S4). The JAK2 V617F allele
AB
burden of patients who did not complete 2 years of treat- ment (n=5) is shown in Online Supplementary Figure S5; two of these patients had reductions in JAK2 V617F allele burden.
Of four patients with a CALR mutation, one had a decrease in allele burden during treatment, two had an increase, and one dropped out of the study before a sec- ond measurement could be done (Online Supplementary Figure S6).
Patients achieving remission after 2 years had a statisti- cally significant greater reduction in JAK2 V617F allele bur- den compared with patients not achieving remission (Figure 2C). Of the 39 patients with the JAK2 V617F muta- tion who completed the study, 15 had a MR and 24 did not; 11 (73%) patients with a MR achieved remission, while five (21%) without a MR achieved remission
C
Figure 2. Change in Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) total symptom score and individual symptoms. (A) Median total symptom score (TSS) with 95% confidence interval (95% CI). (B) The median TSS with 95% CI in participants with remission after 2 years and participants without remission. (C) Individual symptom scores during treatment. Significance is defined as a statistically significant reduction compared with baseline at more than half of the time points. All analyses were done using generalized linear mixed models comparing baseline values with measurements during treatment. *P<0.05; **P<0.01; ***P<0.001
2266
haematologica | 2020; 105(9)