The use of hydroxyurea in the real life of MIOT network: an observational study

ABSTRACT

Background

Hydroxyurea (HU) has been widely used in clinical practice to manage patients with non-transfusion dependent thalassemia (NTDT). Few data are available about the effects of its administration in Italian patients. We assessed hematological and non-hematological outcomes following short- and long-term exposure to HU.

Research design and methods

We considered 71 NTDT patients (30 females) enrolled in the Myocardial Iron Overload in Thalassemia Network and treated for >12 months with HU.

Results

The mean duration of HU treatment was 8.23±5.79 years, starting at a mean age of 37.02±12.06 years. A significant increase in hemoglobin and mean corpuscular volume values and a down-regulation of all erythropoietic and/or hemolysis indices were detected after at least 12 months of treatment. In 28 patients the hemoglobin increase was ≥1.0 g/dl, associated with a higher HU dose. The hematological response dropped in long-term treatment. A favorable impact of HU treatment in limiting the progression of several complications typical of NTDT syndrome was observed.

Conclusion

Our findings seemed to suggest that in several NTDT patients HU could be still a valid option to limit the advance in overall disease clinical burden without carrying significant adverse events and increase in mortality.

KEYWORDS:

• Adverse events
• hemoglobin
• hydroxyurea
• thalassemia

Acknowledgments

We would like to thank all the colleagues involved in the MIOT project (https://miot.ftgm.it/). We thank all patients for their cooperation.

Declaration of interest

A Pepe is the PI of the MIOT project that receives ‘no-profit’ support from Chiesi Farmaceutici S.p.A. and Bayer. A Pepe, A Meloni, and L Pistoia received speakers’ honoraria from Chiesi Farmaceutici S.p.A. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author’s contribution

P Ricchi conceived and designed the study and drafted the initial manuscript. A Meloni analyzed the data and drafted the initial manuscript. P Rigano contributed to the design of the study and collected the data. L Pistoia was responsible for data collection. A Spasiano, M Allò, G Messina, A Quarta, R Rosso, A Quota, A Filosa, and A Maggio collected the data. A Pepe supervised the study and she is the guarantor of this work. All authors assisted with interpretation, commented on drafts of the manuscript, and approved the final version.

Data availability

The data that support the findings of this study are available on request from the corresponding author, A Pepe.

Additional information

Funding

The MIOT project receives ‘no-profit’ support from Chiesi Farmaceutici S.p.A. and Bayer.