Hi Wendy,
I have been waiting for AMG 531/Romiplostim/Nplate since I got my dx May 2006 but since I read an abstract about Romiplostim in MDS patients from ASH 2007 I am waiting for next drug for platelets Eltrombopag/Promacta/Revolade. Here is the abstract:
"Phase 1/2 Study of AMG 531 in Thrombocytopenic Patients (pts) with Low-Risk Myelodysplastic Syndrome (MDS): Update Including Extended Treatment.
Hagop Kantarjian, Pierre Fenaux, Mikkael A. Sekeres, Pamela Becker, Adam Boruchov, David Bowen, Richard Larson, Roger Lyons, Petra Muus, Jamile Shammo, Michael Ehrman, Kuolung Hu, Janet Nichol MD Anderson Cancer Center, Houston, TX, USA; Hopital Avicenne Universite Paris XIII, Bobigny, France; Cleveland Clinic Taussig Cancer Center, Cleveland, OH, USA; Fred Hutchinson Cancer Research Center, Seattle, WA, USA; Memorial Sloan-Kettering Cancer Center, New York, NY, USA; Leeds Teaching Hospitals, Leeds, United Kingdom; University of Chicago, Chicago, IL, USA; Cancer Care Centers South Texas/US Oncology, San Antonio, TX, USA; Radboud University Medical Centre, Nijmegen, Netherlands; Rush University Medical Center, Chicago, IL, USA; Amgen Inc., Thousand Oaks, CA, USA
Background
AMG 531 is a novel thrombopoiesis-stimulating peptibody that is being studied for its ability to increase platelet production by stimulating the thrombopoietin receptor. This report updates outcomes in Part A of the study as of May 2007 on pts who continued into the extension phase of this ongoing phase 1/2, open-label, sequential-cohort, dose-escalation study to evaluate the safety and efficacy of AMG 531 in low risk MDS pts with severe thrombocytopenia.
Methods
Pts with low- risk MDS (IPSS Low or Intermediate-1, excluding CMML), a mean baseline platelet count 50x109/L, and only receiving supportive care were eligible to enter this study.
Pts were enrolled into sequential cohorts of 300, 700, 1000, and 1500g, receiving 3 weekly subcutaneous injections of AMG 531. After evaluation of platelet response at week 4, pts could continue AMG 531 in an optional treatment extension at their assigned dose or dose adjust to achieve or maintain a response.
Results
The mean duration of exposure to AMG 531 was 2315.5 (SD) weeks. Of 44 pts enrolled, 40 continued into the extension phase; 16 pts remain on treatment. Eighteen pts (41%) achieved a durable platelet response (per IWG 2006 criteria for at least 8 consecutive weeks).
Evaluation of durable responses based on baseline platelet count showed that responses occurred in 12/29 (41%) pts with a baseline count of 20x109/L, and in 6/15 (40%) pts with a baseline count of <20x109/L. The mean duration of the platelet response was 22.813.3 (SD) weeks. A total of 104 platelet transfusions were given to 17/44 (39%) pts during this study; of these transfusions, 7 were given in 3/18 (17%) pts who achieved a durable response.
Treatment-related AEs were reported in 17 pts. There were 3 deaths unrelated to treatment. Two confirmed cases of transformation to AML were reported
. These two pts received maximum doses of 300 and 1000g. Six pts were confirmed to have temporary blast cell increases, three of whom had increases above 20%
. Of the 6, 4 were receiving 1500g and 2 were receiving 1000g. In all 6 pts, blast cell counts were observed to have fallen upon follow-up assessments within 7 weeks after treatment discontinuation. In one case, treatment was reinitiated at 700g.
Conclusions
In this study, AMG 531 appeared to be well-tolerated in severely thrombocytopenic low-risk MDS pts, and resulted in increased and sustained platelet counts in the responding pts. AMG 531 may have a role in the treatment of low-risk MDS pts who are thrombocytopenic or have a history of bleeding. These data suggest that further exploration is merited in this pt population. Pt recruitment is ongoing until reaching the planned 84 pts".
Kind regards
Birgitta
68 yo, MDS Interm-1 dx May 2006, transfusion dependent, Desferal 4 days every 5th week, Neupogen 2 injections/week