Contributions
Abstract: P03
Type: Poster presentation
Presentation during EHA Scientific Conference on Bleeding Disorders:
On Friday, September 16, 2016 from 14:00 - 15:30
Location: Cristal + Coral
Background
The thrombopoietin receptor agonist romiplostim is approved for use in adults with chronic ITP. In this study, patients with ITP (N=169) had bone marrow biopsies performed at baseline and after 1, 2, or 3 years of romiplostim; 24 patients discontinued romiplostim and entered remission.
Aims
To examine remission in patients with ITP receiving romiplostim in a bone marrow study.
Methods
Patients with ITP entering the bone marrow study had a platelet count <50×109/L and ≥1 prior ITP therapy. Romiplostim, received weekly for up to 3 years, was adjusted from 1 10 μg/kg to target platelet counts of 50-200×109/L; doses were reduced for platelet counts >200×109/L for 2 consecutive weeks and no romiplostim was given for platelet counts >400×109/L. A post hoc analysis was conducted of those who entered remission, ie platelet counts ≥50×109/L for ≥6 months with no ITP therapy, including romiplostim.
Results
The median years since ITP diagnosis for those who did (N=24) and did not (N=145) enter remission (1.66 years vs 5.16 years) had overlapping ranges [not significant (NS)], as did the median average weekly dose (1.1 μg/kg vs 3.5 μg/kg, NS, included zero doses prior to last non-zero dose) (Table). Adverse events (AEs) occurred at similar rates. A post hoc analysis examining the association between remission and baseline factors including age, gender, platelet count, prior splenectomy, ITP duration, and number of prior treatments indicated that ITP duration ≤1 year could be a potential predictor for remission (hazard ratio 2.46, 95% CI: 1.04, 5.79, p=0.04); however, this association could be due to multiple comparisons (ie, type I error). Median time of onset for remission was 52 weeks (range, 6–124 weeks) and median duration of remission during the study was 88 weeks (range, 29–154 weeks); 21 of the 24 patients were still in remission at the last observation on study.
Conclusion/Summary
In this post hoc analysis, 14% (24/169) of patients in a romiplostim ITP bone marrow study were able to enter remission following standard dosing rules; more studies are needed to confirm whether shorter ITP duration is a predictor of remission.
| Characteristic | Remission (N=24) | No Remission (N=145) |
| Women, n (%) | 12 (50) | 102 (70) |
| Age, y, median (Q1, Q3) | 45.5 (31.0, 58.0) | 51.0 (37.0, 64.0) |
| Years since ITP diagnosis, median (Q1, Q3) | 1.66 (0.46, 7.75) | 5.16 (1.62, 12.84) |
| Prior splenectomy, n (%) | 7 (29.2) | 53 (36.6) |
| Platelet count at screening, ×109/L, median (Q1, Q3) | 20.9 (7.5, 35.0) | 23.0 (11.0, 35.0) |
| Time to first platelet response, n, weeks, median (Q1, Q3) | 24, 3.5 (2.0, 14.0) | 131, 2.0 (2.0, 6.0) |
| Treatment duration, weeks, median (Q1, Q3) | 62.5 (14.1, 82.1) | 155.7 (66.0, 156.0) |
| Average weekly dose, μg/kg, median (Q1, Q3) | 1.1 (1.0, 2.2) | 3.5 (1.9, 7.2) |
| Splenectomy on study, n (%) | 0 | 3 (2.1) |
| Any treatment-related AE, n (%) | 9 (37.5) | 51 (35.2) |
| Serious AE / Treatment-related serious AE, n (%) | 8 (33.3) / 0 | 48 (33.1) / 6 (4.1) |
| Fatal AE, n (%) | 0 | 7 (4.8) |
| Bone marrow changes (increased reticulin ≥2 grades or collagen), n (%) | 0 | 9 (6.2) |
| On-study bleeding AE, n (%) | 15 (62.5) | 84 (57.9) |
| On-study grade ≥2 bleeding AE, n (%) | 4 (16.7) | 34 (23.5) |
| On-study serious bleeding AE, n (%) | 1 (4.2) | 13 (9.0) |
Abstract: P03
Type: Poster presentation
Presentation during EHA Scientific Conference on Bleeding Disorders:
On Friday, September 16, 2016 from 14:00 - 15:30
Location: Cristal + Coral
Background
The thrombopoietin receptor agonist romiplostim is approved for use in adults with chronic ITP. In this study, patients with ITP (N=169) had bone marrow biopsies performed at baseline and after 1, 2, or 3 years of romiplostim; 24 patients discontinued romiplostim and entered remission.
Aims
To examine remission in patients with ITP receiving romiplostim in a bone marrow study.
Methods
Patients with ITP entering the bone marrow study had a platelet count <50×109/L and ≥1 prior ITP therapy. Romiplostim, received weekly for up to 3 years, was adjusted from 1 10 μg/kg to target platelet counts of 50-200×109/L; doses were reduced for platelet counts >200×109/L for 2 consecutive weeks and no romiplostim was given for platelet counts >400×109/L. A post hoc analysis was conducted of those who entered remission, ie platelet counts ≥50×109/L for ≥6 months with no ITP therapy, including romiplostim.
Results
The median years since ITP diagnosis for those who did (N=24) and did not (N=145) enter remission (1.66 years vs 5.16 years) had overlapping ranges [not significant (NS)], as did the median average weekly dose (1.1 μg/kg vs 3.5 μg/kg, NS, included zero doses prior to last non-zero dose) (Table). Adverse events (AEs) occurred at similar rates. A post hoc analysis examining the association between remission and baseline factors including age, gender, platelet count, prior splenectomy, ITP duration, and number of prior treatments indicated that ITP duration ≤1 year could be a potential predictor for remission (hazard ratio 2.46, 95% CI: 1.04, 5.79, p=0.04); however, this association could be due to multiple comparisons (ie, type I error). Median time of onset for remission was 52 weeks (range, 6–124 weeks) and median duration of remission during the study was 88 weeks (range, 29–154 weeks); 21 of the 24 patients were still in remission at the last observation on study.
Conclusion/Summary
In this post hoc analysis, 14% (24/169) of patients in a romiplostim ITP bone marrow study were able to enter remission following standard dosing rules; more studies are needed to confirm whether shorter ITP duration is a predictor of remission.
| Characteristic | Remission (N=24) | No Remission (N=145) |
| Women, n (%) | 12 (50) | 102 (70) |
| Age, y, median (Q1, Q3) | 45.5 (31.0, 58.0) | 51.0 (37.0, 64.0) |
| Years since ITP diagnosis, median (Q1, Q3) | 1.66 (0.46, 7.75) | 5.16 (1.62, 12.84) |
| Prior splenectomy, n (%) | 7 (29.2) | 53 (36.6) |
| Platelet count at screening, ×109/L, median (Q1, Q3) | 20.9 (7.5, 35.0) | 23.0 (11.0, 35.0) |
| Time to first platelet response, n, weeks, median (Q1, Q3) | 24, 3.5 (2.0, 14.0) | 131, 2.0 (2.0, 6.0) |
| Treatment duration, weeks, median (Q1, Q3) | 62.5 (14.1, 82.1) | 155.7 (66.0, 156.0) |
| Average weekly dose, μg/kg, median (Q1, Q3) | 1.1 (1.0, 2.2) | 3.5 (1.9, 7.2) |
| Splenectomy on study, n (%) | 0 | 3 (2.1) |
| Any treatment-related AE, n (%) | 9 (37.5) | 51 (35.2) |
| Serious AE / Treatment-related serious AE, n (%) | 8 (33.3) / 0 | 48 (33.1) / 6 (4.1) |
| Fatal AE, n (%) | 0 | 7 (4.8) |
| Bone marrow changes (increased reticulin ≥2 grades or collagen), n (%) | 0 | 9 (6.2) |
| On-study bleeding AE, n (%) | 15 (62.5) | 84 (57.9) |
| On-study grade ≥2 bleeding AE, n (%) | 4 (16.7) | 34 (23.5) |
| On-study serious bleeding AE, n (%) | 1 (4.2) | 13 (9.0) |