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Case Reports
. 2022 Dec 9;10(12):3201.
doi: 10.3390/biomedicines10123201.

Case Report of Myelodysplastic Syndrome in a Sickle-Cell Disease Patient Treated with Hydroxyurea and Literature Review

Pagona Flevari  1 , Ersi Voskaridou  1 , Frédéric Galactéros  2 , Giovanna Cannas  3 , Gylna Loko  4 , Laure Joseph  5 , Pablo Bartolucci  2 , Justine Gellen-Dautremer  2 , Emmanuelle Bernit  6 , Corine Charneau  6 , Anoosha Habibi  2
Affiliations
Case Reports

Case Report of Myelodysplastic Syndrome in a Sickle-Cell Disease Patient Treated with Hydroxyurea and Literature Review

Pagona Flevari et al. Biomedicines. .

Abstract

The safety profile of hydroxyurea (HU) in patients with sickle-cell disease (SCD) is relatively well known. However, despite the suspected association of HU with myeloid neoplasms in myeloproliferative neoplasms (MPN), and the publication of sporadic reports of myeloid malignancies in SCD patients treated with HU, the possible excess risk imparted by HU in this population having an increasing life expectancy has failed to be demonstrated. Herein, we report one case of myelodysplastic syndrome emanating from the results on safety and effectiveness of HU on the largest European cohort of 1903 HU-treated adults and children who were followed-up prospectively in an observational setting over 10 years, accounting for a total exposure of 7309.5 patient-years. A comparison of this single case with previously published similar cases did not allow us to draw any significant conclusions due to the paucity of these events.

Keywords: case report; hydroxyurea; myelodysplastic syndrome; sickle cell disease.

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Conflict of interest statement

Ersi Voskaridou reported receiving fees from CELGENE (Consultancy, Research funding), Bristol Myers Squibb (BMS) (Consultancy, Research funding), PROTAGONIST (Research funding), GENESIS (Consultancy, Research funding), NOVARTIS (Research funding), ADDMEDICA (Consultancy, Research funding), and IMARA (Research funding). Frédéric Galactéros reported receiving grants for consultancy from NOVARTIS, SANOFI, BLUEBIRDBIO, PFIZER, AGIOS, GBT, BMS and ADDMEDICA (Consultancy, Research funding). Giovanna Cannas reported receiving fees from ADDMEDICA (Research funding). Laure Joseph reported receiving fees from BLUEBIRDBIO (Consultancy), and ADDMEDICA (Research funding). Pablo Bartolucci reported receiving fees from NOVARTIS Company (Consultancy, Lecture, Research funding), BLUEBIRDBIO (Consultancy, Research funding), EMMAUS (Consultancy), HEMANEXT (Consultancy), AGIOS (Consultancy), JAZZPHARMA (Lecture), ADDMEDICA (Consultancy, Research funding), FOUNDATION FABRE (Research funding), Cofounder of INNOVHEM, ROCHE (Consultancy), and GBT (Consultancy). Justine Gellen-Dautremer reported receiving fees from ADDMEDICA (Research funding). Emmanuelle Bernit reported receiving fees from ADDMEDICA (Research funding). Anoosha Habibi reported receiving fees from NOVARTIS Company (Consultancy), BLUEBIRD BIO Company (Consultancy), and ADDMEDICA (Research funding). Addmedica Company was involved in the design and conduct of the study (including development of the study protocol and statistical analysis plan); collection, monitoring, analysis, and interpretation of the data; and preparation, approval and submission of this manuscript.

Figures

Figure 1
Figure 1
Causes of death in HU and non-HU patients (adapted from [10]).
Figure 2
Figure 2
Probability of 10-year overall survival in HU SCD patients versus non-HU SCD patients (adapted from [10]).
See this image and copyright information in PMC

References

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