THE FINAL ANALYSIS OF EXPAND: A PHASE 1B, OPEN-LABEL, DOSE-FINDING
STUDY OF RUXOLITINIB IN PATIENTS WITH MYELOFIBROSIS AND LOW
POSTER 9
PLATELET COUNT (50 × 109/L TO < 100 × 109/L) AT BASELINE
Paola Guglielmelli1, Jean-Jacques Kiladjian2, Alessandro M. Vannucchi3, Minghui Duan4, Haitao Meng5,
Ling Pan6, Guangsheng He7, Srdan Verstovsek8, Françoise Boyer-Perrard9, Fiorenza Barraco10, Dietger
Niederwieser11, Ester Pungolino12, Anna Marina Liberati13, Claire Harrison14, Pantelia Roussou15,
Monika Wroclawska15, Kevin Majidi15, Peter A.W. te Boekhorst16 and Heinz Gisslinger17
Dr. te Boekhorst and Dr Gisslinger contributed equally to this analysis.
(1)CRIMM-Centro Ricerca e Innovazione delle Malattie Mieloproliferative, Azienda Ospedaliera–
Universitaria Careggi, University of Florence, Florence, Italy
(2)Hôpital Saint-Louis & Université de Paris, Paris, France
(3)CRIMM, Center for Research and Innovation of Myeloproliferative Neoplasms, AOU Careggi,
Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy
(4)Peking Union Medical College Hospital, Beijing, China
(5)Department of Hematology, the First Affiliated Hospital, College of Medicine, Zhejiang University;
Institute of Hematology, Zhejiang University, Hangzhou, China
(6)West China Hospital of Sichuan University, Chengdu, China
(7)Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu
Province Hospital, Nanjing, China
(8)University of Texas MD Anderson Cancer Center, Houston, TX, USA
(9)CHU Angers, Angers, France
(10)Centre Hospitalier Lyon Sud, Pierre-Bénite, France
(11)Medical Clinic and Policlinic 1, Hematology and Cellular Therapy, Leipzig University Hospital,
Leipzig, Germany
(12)Niguarda Ca' Granda Hospital, Milan, Italy
(13)Azienda Ospedaliera "Santa Maria", Terni, Italy
(14)Department of Haematology, Guy’s and St. Thomas’ NHS Foundation Trust, London, United
Kingdom
(15)Novartis Pharma AG, Basel, Switzerland
(16)Erasmus Medical Center, Rotterdam, Netherlands
(17)Department of Internal Medicine I, Division of Hematology & Hemostaseology, Medical University
of Vienna, Vienna, Austria
Background: Ruxolitinib is a JAK1/JAK2 inhibitor shown to improve splenomegaly, symptom
burden, and survival in patients with myelofibrosis (MF). Despite the high incidence of
thrombocytopenia in MF, the phase 3 COMFORT trials assessing ruxolitinib in patients with
MF did not include patients with platelet counts <100 x 109/L. Here, we present the final
results from EXPAND, which evaluated the safety and tolerability of ruxolitinib in patients with
low platelet counts at baseline. Results from previous analyses established the maximum safe
starting dose (MSSD) at 10 mg bid.
Methods: EXPAND was a phase 1b, open-label, dose-finding study of ruxolitinib in patients
with MF and baseline platelet count of 50 to <100 × 109/L (NCT01317875). Patients were
stratified according to platelet count into stratum 1 (S1, 75 to <100 × 109/L) and stratum 2 (S2,
50 to <75 × 109/L). The study included a dose escalation and safety period (up to week 24) to
establish the MSSD, and a safety expansion period (from week 24 up to 3 years). The primary
SCIENTIFIC PROGRAMME
SESSION I
OPTIMIZING
CYTOREDUCTION
SESSION II
MANAGEMENT OF CML
WITH TKI
SESSION III
MPN RISK
STRATIFICATION
INCLUDING VASCULAR
EVENTS
DEBATE 1
INTERFERON ALPHA
SHOULD BE FRONT LINE
THERAPY IN ALL ET/PV
PATIENTS
ROUNDTABLE 1
INFECTIONS IN
MYELOPROLIFERATIVE
DISORDERS, INCLUDING
CML
ROUNDTABLE 2
PREGNANCY AND
PARENTING
DEBATE 2
ALLOGENEIC STEM CELL
TRANSPLANTATION
SHOULD BE CONSIDERED
THIRD LINE OPTION IN
CHRONIC PHASE CML
SESSION IV
EVOLVING THERAPIES
IN MYELOFIBROSIS
SESSION V
MANAGEMENT OF
ADVANCED AND UNUSUAL
DISEASE (MPN AND CML)
SESSION VI
TREATMENT FREE
REMISSION IN CML
KEYNOTE LECTURE
SELECTED ABSTRACTS
FOR AN ORAL
PRESENTATION
SELECTED ABSTRACTS
FO R A POSTER
PRESENTATION
DISCLOSURES