POSTER 29
TRIPLE-NEGATIVE PRIMARY MYELOFIBROSIS WITH GRADE 3
THROMBOCYTOPENIA SUCCESSFULLY MANAGED WITH LOW DOSE
RUXOLUTINIB
Hugo Verdelho Parreira, Mariana Leal Fernandes, Paula Sousa e Santos and Patrícia Ribeiro
Serviço de Hematologia Clínica, Hospital dos Capuchos, Centro Hospitalar e Universitário Lisboa
Central, Lisboa, Portugal
Background: The management of primary myelofibrosis (PMF) remains a challenge,
particularly in profound cytopenic patients. Ruxolitinib, a JAK2 inhibitor, improves
splenomegaly and disease associated symptoms. However, thrombocytopenia (that occurs in
¼ of these patients) frequently limits the use of the drug. Recent data has shown that a dose
reduction might be safe and effective, but is still regarded with caution when the platelet
count is below 50x10^9/L.
Case Report: A 77 years old male patient referred to our hematology department in 2018 due
to bicytopenia, diagnosed with triple-negative PMF DIPSS-Plus score of 4 (high risk). At the
time of diagnosis, the patient presented with symptomatic anemia requiring transfusion.
Anemia and thrombocytopenia worsened and the patient became more dependent on
transfusion support, with an average need of one red cell unit per month. He also developed
constitutional symptoms (fatigue and night sweats), along with palpable splenomegaly.
Although he had a platelet count between 34-43×10^9/L, we decided to start Ruxolutinib at
the dose of 5mg twice daily. He became gradually asymptomatic and achieved transfusion
independence 7 months after the start of the drug. The patient has now been maintained on
the same dose of Ruxolutinib for 11 months, with improvement of his general quality of life
and a consistent improvement in the patient blood counts, with current Hb of 9.4g/dL and
platelets of 180x10^9/L. We have decided to maintain Ruxolutinib 5mg twice daily in light of
these favorable results.
Conclusions: The patient has a high risk JAK2 negative myelofibrosis and, albeit current
literature do not recommend starting Ruxolutinib on thrombocytopenic patients with a
platelet count below 50x10^9/L, we demonstrated it is an appropriate option in this case.
Severe thrombocytopenia is frequently both a consequence and a limitation for the use of
JAK2 inhibitors in myelofibrosis and, despite lacking more robust evidence, some previous
data supports our option for the use of Ruxolutinb low dose as an effective and safe therapy
in this clinical setting.
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