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Multicenter Study
. 2021 Jul;194(2):336-343.
doi: 10.1111/bjh.17539. Epub 2021 Jun 20.

Eltrombopag for myelodysplastic syndromes or chronic myelomonocytic leukaemia with no excess blasts and thrombocytopenia: a French multicentre retrospective real-life study

Thibault Comont  1 , Mathieu Meunier  2 , Amina Cherait  3 , Clemence Santana  4 , Thomas Cluzeau  5 , Bohrane Slama  6 , Kamel Laribi  7 , Jean-Thomas Giraud  8 , Sophie Dimicoli  9 , Ana Berceanu  10 , Lenaïg Le Clech  11 , Pascale Cony-Makhoul  12 , Berangere Gruson  13 , Jose Torregrosa  14 , Laurence Sanhes  15 , Vincent Jachiet  16 , Marie-Agnes Azerad  17 , Ahmad Al Jijakli  18 , Emmanuel Gyan  19 , Clement Gaudin  20 , Jonathan Broner  21 , Claire Guerveno  22 , Thierry Guillaume  23 , Pr Lionel Ades  3 , Odile Beyne-Rauzy  1 , Pierre Fenaux  3 ; Groupe Francophone des Myélodysplasies (GFM)
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Free article
Multicenter Study

Eltrombopag for myelodysplastic syndromes or chronic myelomonocytic leukaemia with no excess blasts and thrombocytopenia: a French multicentre retrospective real-life study

Thibault Comont et al. Br J Haematol. 2021 Jul.
Free article

Abstract

Despite a moderate prevalence in low-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukaemia (CMML), thrombocytopenia remains a risk of severe bleeding and therapeutic options are still limited. There are only a few studies with eltrombopag (ELT), a thrombopoietin receptor agonist, in those patients. In this retrospective multicentre study, ELT was used in 50 patients with MDS and 11 with CMML, with no excess of marrow blasts and platelet counts of <50 × 109 /l in a 'real-life' situation. Platelet response occurred in 47 (77%) patients. The median (range) duration of response was 8 (0-69) months. None of the eight still responders who discontinued ELT had relapsed, at a median (range) of 16 (6-23) months after ELT discontinuation. Although 36% of the patients were anti-coagulated or anti-aggregated only 10% of patients had Grade ≥3 bleeding events. Thrombotic events were observed in six (10%) patients, who all but one had a medical history of arterial or venous thrombosis. Progression to acute myeloid leukaemia occurred in four (7%) patients. In this first 'real-life' study, ELT was effective and generally well tolerated in patients with MDS/CMML without excess blasts.

Keywords: chronic myelomonocytic leukaemia; eltrombopag; myelodysplastic syndromes; real-life; thrombocytopenia.

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References

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