limit of detection. Overall, 85% of patients had stability in the grading of their esophageal
varices; 13/21 patients had a 50% reduction in spleen size by palpation and 6 patients had
spleen volume reduction of at least 35%, confirmed by imaging. There were no unexpected
safety concerns.4
In this case vignette, with HU intolerance, relative contraindications to approved therapies,
and a patient preference, ruxolitinib becomes a reasonable option. Use here is individualized
and empirical, as studies have not demonstrated improvement in hard endpoints. A clinical
trial would also be suitable for such a patient, and novel agents on the horizon include
ropeginterferon and bomedemstat.
References:
1. Barbui, T., et al., Practice-relevant revision of IPSET-thrombosis based on 1019 patients with
WHO-defined essential thrombocythemia. Blood Cancer J, 2015. 5: p. e369.
2. Verstovsek, S., et al., Ruxolitinib for essential thrombocythemia refractory to or intolerant of
hydroxyurea: long-term phase 2 study results. Blood, 2017. 130(15): p. 1768-1771.
3. Harrison, C.N., et al., Ruxolitinib vs best available therapy for ET intolerant or resistant to
hydroxycarbamide. Blood, 2017. 130(17): p. 1889-1897.
4. Pieri, L., et al., Safety and efficacy of ruxolitinib in splanchnic vein thrombosis associated with
myeloproliferative neoplasms. Am J Hematol, 2017. 92(2): p. 187-195.
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