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A retrospective study of azacitidine treatment in patients with intermediate-2 or high risk myelodysplastic syndromes in a real-world clinical setting in Greece

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Abstract

For patients with intermediate-2 or high risk [according to the International Prognostic Scoring System (IPSS)] myelodysplastic syndromes (MDS), azacitidine treatment offers hematologic improvement (HI) but also has the potential to modify the natural disease course. ‘RETRO-AZA-MDS-001’, a retrospective chart review study was conducted from February to November 2012 across 17 hematology hospital sites of Greece, aiming to evaluate the clinical efficacy and safety profile of azacitidine in IPSS intermediate-2/high risk adult MDS patients in routine care. A total of 88 patients (median age 74.7 years), with a 6.6 month median (range 1.0–49.5) azacitidine treatment duration were enrolled. The overall response rate [complete response (CR), marrow CR and partial response] was 37.7% (23/61), while stable disease with HI was achieved by 21.3% (13/61). The HI rate was 33.0 % (29/88) and the AML transformation rate 6.8% (6/88). Of the transfusion-dependent patients, 7.3% (3/41) became transfusion-independent during azacitidine treatment. The incidence of non-serious and serious adverse events related to azacitidine was 50.0 and 42.0%, respectively. Patients not receiving prior ESA therapy were expected to be 7.6 times more likely to achieve a clinical response (p = 0.012). The study corroborates the favorable risk–benefit profile of azacitidine for intermediate-2/high risk MDS patients in routine clinical practice.

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Abbreviations

ADR:

Adverse drug reaction

allo-SCT:

Allogeneic stem cell transplantation

AML:

Acute myeloid leukemia

BMI:

Body mass index

CI:

Confidence interval

CMML:

Chronic myelomonocytic leukemia

CR:

Complete response

CRF:

Case record form

CTC:

Common toxicity criteria

ESA:

Erythropoiesis-stimulating agent

G-CSF:

Granulocyte-colony stimulating factor

HI:

Hematologic improvement

ICF:

Informed consent form

IPSS:

International Prognostic Scoring System

IWG:

International Working Group

mCR:

Marrow CR

MDS:

Myelodysplastic syndromes

NSADR:

Non-serious drug adverse reaction

OIR:

Overall improvement rate

OR:

Odds ratio

ORR:

Overall response rate

OS:

Overall survival

PR:

Partial response

RAEB-1:

Refractory anemia with excess blasts type 1 (5–9% blasts in bone marrow)

RAEB-2:

Refractory anemia with excess blasts type 2 (10–19% blasts in bone marrow)

RAEB-t:

Refractory anemia with excess blasts in transformation

RBC:

Red blood cell

RCMD:

Refractory cytopenia with multilineage dysplasia

RCUD:

Refractory cytopenia with unilineage dysplasia

SADR:

Serious adverse drug reaction

SAE:

Serious adverse event

SD:

Standard deviation

WHO:

World Health Organisation

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Acknowledgements

The authors wish to thank Qualitis Ltd. for study monitoring and data analysis which was funded by GENESIS Pharma SA.

Author information

Authors and Affiliations

  1. University General Hospital ‘Attikon’, 1, Rimini Str, Chaidari, 12462, Athens, Greece

    Vasiliki Pappa

  2. General Hospital of Thessaloniki ‘G. Papanikolaou’, Eksoxi, 57010, Thessaloniki, Greece

    Achilles Anagnostopoulos

  3. University General Hospital of Larissa, Mezourlo, 41110, Larissa, Greece

    Eleni Bouronikou

  4. University Hospital of Ioannina, 45100, Ioannina, Greece

    Evangelos Briasoulis

  5. Department of Hematology, Medical School, Democritus University of Thrace, Xanthi, Greece

    Ioannis Kotsianidis

  6. General Hospital of Athens ‘Evaggelismos’, Ipsilantou 45, 10676, Athens, Greece

    Maria Pagoni

  7. General Hospital of Patras ‘Agios Andreas’, Tsertidou 1, 26335, Patras, Greece

    Panagiotis Zikos

  8. ‘251’ General Airforce Hospital, Kanellopoulou 3, 11525, Athens, Greece

    Konstantinos Tsionos

  9. ‘Laiko’ General Hospital of Athens, Agiou Thoma 17, 11527, Athens, Greece

    Nora Viniou & John Meletis

  10. University General Hospital of Heraklion, Voutes, 71110, Heraklion, Greece

    Helen Papadaki

  11. General Hospital of Thessaloniki ‘Papageorgiou’, Circular Road Neas Efkarpias, 56429, Thessaloniki, Greece

    Anna Kioumi

  12. General Hospital of Athens ‘G. Gennimatas’, Mesogeion 154, 11527, Athens, Greece

    Athanasios Galanopoulos

  13. ‘Henry Dunant’ Hospital, 107 Mesogeion Av, 11526, Athens, Greece

    Elisavet-Christine Vervessou

  14. ‘401’ Army General Hospital of Athens, Mesogeion and Kanellopoulou 1, 11525, Athens, Greece

    Elias Poulakidas

  15. Army Share Fund Hospital ‘NIMTS’, Monis Petraki 10, 11521, Athens, Greece

    Panagiotis Karmas

  16. GENESIS Pharma SA, 270 Kifisias Av, 15232, Athens, Greece

    Kiki Karvounis

  17. General University Hospital of Patras, Rio Patron, 26500, Patras, Greece

    Argiris Symeonidis

Authors
  1. Vasiliki Pappa
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  2. Achilles Anagnostopoulos
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  3. Eleni Bouronikou
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  4. Evangelos Briasoulis
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  5. Ioannis Kotsianidis
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  6. Maria Pagoni
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  7. Panagiotis Zikos
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  8. Konstantinos Tsionos
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  9. Nora Viniou
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  10. John Meletis
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  11. Helen Papadaki
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  12. Anna Kioumi
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  13. Athanasios Galanopoulos
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  14. Elisavet-Christine Vervessou
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  15. Elias Poulakidas
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  16. Panagiotis Karmas
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  17. Kiki Karvounis
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  18. Argiris Symeonidis
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Corresponding author

Correspondence to Vasiliki Pappa.

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Conflict of interest

The study was funded by GENESIS Pharma SA, Greece. Kiki Karvounis is an employee of GENESIS Pharma SA. The remaining authors have no conflicts of interest to declare.

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Pappa, V., Anagnostopoulos, A., Bouronikou, E. et al. A retrospective study of azacitidine treatment in patients with intermediate-2 or high risk myelodysplastic syndromes in a real-world clinical setting in Greece. Int J Hematol 105, 184–195 (2017). https://doi.org/10.1007/s12185-016-2115-y

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  • DOI: https://doi.org/10.1007/s12185-016-2115-y

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