Effectiveness and safety of 5-azacitidine in three patients with myelodysplastic syndromes

Garzás-Martín de Almagro, M.C.; Gago Sánchez, A.I.; Cuevas Asencio, I.; Reyes Malia, M.

doi:10.1016/S2173-5085(10)70014-9

Farmacia Hospitalaria

ISSN: 1130-6343

Farmacia Hospitalaria (Farm Hosp) is the official publication of the Spanish Society of Hospital Pharmacists (SEFH), a professional and scientific organization that currently has about 4.351 members.

The Mission of Farm Hosp is to be the support for health professionals for expanding the influence of projects, publications, and training programs, and to promote the exchange of experiences.

Vision Farm Hosp is:
- to increase the state of knowledge about the hospital pharmacy and whose actions should increase the adequate and safe use of medicines and health products.
- to be an instrument of exchange and dissemination of scientific knowledge of pharmacy practice through the best use and safe medication and health outcomes.

It is a peer-reviewed double-blind scientific journal aimed at publishing in the broad field of Hospital Pharmacy advances in Pharmacy Services. This journal included the following topics: Drug evaluation. Quality of life. Patient Reported Outcome Measures (PROMs), and Patient Reported Experience Measures (PREMs) in the use of drugs. Monitoring medication. Pharmaceutical compounding. Medication administration. Management of Pharmacy Services (logistics, quality control). Technologies and information systems in the process of using medication. Medical devices. Patient Education. Pharmacist´s role in the society and /or in the Health System in Spain, and other topics related to clinical, scientific, and institutional role of hospital pharmacists.

Farmacia Hospitalaria publishes bimonthly (6 issues yearly) originals, short originals, reviews, clinical cases, special articles, protocols, consensus documents, clinical guidelines, letters to the editor, editorials.

All the articles are published in Spanish and English language regardless of the language in which they are received.

Farmacia Hospitalaria is an Open Access journal, whose content is available free of charge to users or their institutions. Users are authorised to read, download, copy, distribute, print, search, or link to entire texts of the articles in this journal without prior permission from the editor or author, according to the BOAI definition of Open Access. The papers may be re-used under the terms of the Creative Commons 4.0 license. (CC BY-NC-ND). Such use shall be subject to authorisation from SEFH, the journal owner.

Farmacia Hospitalaria journal charges no fees for submission of papers or publication of articles.

Adherence to international ethics recommendations

Manuscripts must follow the most recent recommendations of the International Committee of Medical Journal Editors (available at http://www.icmje.org).

The articles published in Farmacia Hospitalaria are indexed in PubMed and other 17 date bases:
- Evaluation databases: MEDLINE, DOAJ, Scopus, Latindex y Scimago Journal and Country Rank (SJR). Índice H: 18, quartile 3 (Medicine, miscellaneous / Pharmacology)
- Bibliographic databases: CINHAL, EMBASE Excerpta Medica, Índice Bibliográfico Español en Ciencias de la Salud (IBECS), Índice Español de Ciencia Y Tecnología (ICyT), Índice Médico Español (IME), International Pharmaceutical Abstracts (IPA).
- Journal collections: Dialnet, Medes, Scientific Electronic Library Online (SciELO), REDIB, ScienceDirect, ClinicalKey.

Indexed in:

Medline, Emerging Sources Citation Index (ESCI), Directory of Open Access Journals (DOAJ)

Review of medical records of patients who received 5-azacitidine 75mg/m2 subcutaneously for during 7 days every 28 days in twelve cycles as compassionate use. We evaluated the objective response, clinical improvement and time to disease progression. We recorded adverse reactions described in the medical history.

Results

Six patients were candidates for treatment with 5-azacitidine. Three cases were evaluated over the study period. Most remained in partial response or better after the study, and no longer needed transfusions. In one patient, the treatment appeared to delay progression to leukaemia.

Conclusions

5-Azacitidine might be considered an effective and relatively safe drug, and may have contributed to controlling peripheral cytopenias, improving the quality of life and delaying progression to leukaemia. Additional studies with more patients are needed to support these results.

Keywords:

5-azacitidine

Myelodysplastic syndrome

Effectiveness

Safety

Quality of life

Resumen

Objetivo

Evaluar la efectividad y seguridad del tratamiento con 5-azacitidina en síndrome mielodisplásico.

Métodos

Revisión de historias clínicas de pacientes que recibieron 5-azacitidina 75mg/m2 subcutánea durante 7 días, cada 28 días en 12 ciclos. Se valoró la respuesta objetiva, mejoría clínica y tiempo hasta la progresión de la enfermedad. Se recogieron las reacciones adversas descritas en la historia clínica.

Resultados

Seis pacientes fueron candidatos a tratamiento con 5-azacitidina. Tres casos fueron evaluables tras el período considerado. La mayoría permanecieron en respuesta parcial o mejor al finalizar el estudio, dejando de precisar transfusiones. En una paciente se retrasó la progresión a leucemia.

Conclusiones

5-Azacitidina podría considerarse un fármaco relativamente efectivo y seguro, pudiendo haber contribuido al control de citopenias periféricas, a mejorar la calidad de vida y a retrasar la progresión a leucemia. Serían necesarios estudios con mayor número de pacientes que corroborasen estos resultados.

Palabras clave:

5-Azacitidina

Síndrome mielodisplásico

Efectividad

Seguridad

Calidad de vida

Full text is only available in PDF

References

[1.]

M.L. Heaney, D.W. Golde.

Myelodisplasia.

N Engl J Hematol, 340 (1999), pp. 1649-1660

[2.]

P.L. Greenberg, N.S. Young, N. Gatterman.

Myelodysplastic syndromes.

American Society of Hematology Meeting, (2002), pp. 136-161

[3.]

E.H. Estey.

Current challenges in therapy of myelodysplastic syndromes.

Curr Opin Hematol, 10 (2003), pp. 60-67

Medline

[4.]

M. Sullivan, K. Hahn, J. Kolesar.

Azacitidine: A novel agent for myelodisplastic síndromes.

Am J Health-Syst Pharm, 62 (2005), pp. 1567-1573

http://dx.doi.org/10.2146/ajhp040385 | Medline

[5.]

National Comprehensive Cancer Network. Practice guidelines in oncology: myelodysplastic syndromes v.2.2009.(http://www.nccn.org/professionals/physicians_gls/PDF/mds.pdf).

[6.]

J. Meletis, N. Viniou, E. Terpos.

Novel agents for the management of myelodysplastic síndromes.

Med Sci Monit, 12 (2006), pp. RA194-RA2006

Medline

[7.]

L.R. Silverman, E.P. Demakos, B.L. Peterson, et al.

Randomized controlled trial or azacitidine in patients whith the myelodysplastic syndromes: a study of the Cancer and Leukemia Group B.

J Clin Oncol, 20 (2002), pp. 2429-2440

http://dx.doi.org/10.1200/JCO.2002.04.117 | Medline

[8.]

L.R. Silverman, J.F. Holland, E.P. Demakos, et al.

Azacitidine (Aza C) in myelodysplastic syndromes (MSD) CALGB studies 8421 and 8921.

Ann Hematol, 68 (1994), pp. A12

[9.]

L.R. Silverman, J.F. Holland, R.R. Ellison.

Low dose continous infusion azacytidine is an effective therapy for patients with myelodysplastic syndromes, a study of the Cancer and Leukemia Group B.

J Cancer Res Clin Oncol, 116 (1990), pp. 816

[10.]

M.D. Haifa Abdulhaq, M. James, D.O. Rossetti.

The role of azacitidine in the treatment of myelodysplastic syndromes.

Expert Opin Investig Drugs, 16 (2007), pp. 1967-1975

http://dx.doi.org/10.1517/13543784.16.12.1967 | Medline

[11.]

Cancer Therapy Evaluation Program, Common Terminology Criteria for Adverse Events, Version 3.0, DCTD, NCI, NIH, DHHS. March 31, 2003 (http://www.ctep.cancer.gov). Publish date: August 9, 2006.

[12.]

E. Kaminskas, A.T. Farell, Y.C. Wang, R. Sridhara, R. Pazdur.

FDA drug approval summary: Azacitidine (5-Azacydtidine Vidaza TM) for injectable suspension.

Oncologist, 10 (2005), pp. 176-182

http://dx.doi.org/10.1634/theoncologist.10-3-176 | Medline

[13.]

L.R. Silverman, D.R. McKenzie, B.L. Peterson, J.F. Hollan, J.T. Backstrom, C.L. Beach, Cancer and Leukemia Group, et al.

Further of trials with azacitidine in patiens with myelodysplastic syndrome: studies 8421, 8921 and 9221 by the Cancer and Leukemia Group.

J Clin Oncol, 24 (2006), pp. 3895-3903

[14.]

A.B. Kornblith, J.E. Herndón, L.R. Silverman, E.P. Demakos, R. Odchimar-Reissig, J.F. Holland, et al.

Impact of azacitidine of life of patients with myelodysplastic syndrome treated in a randomized phase III trial: a Cancer and Leukemia Group B Study.

J Clin Oncol, 20 (2002), pp. 2441-2452

http://dx.doi.org/10.1200/JCO.2002.04.044 | Medline

[15.]

P. Fenaux, G.J. Mufti, E. Hellstrom-Lindberg, V. Santini, C. Finelli, A. Giagounidis, et al.

Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher risk myelodysplastic syndromes: a randomised, open-label, phase III study.

Lancet Oncol, 10 (2009), pp. 223-232

Tools

Indexed in:

Follow us:

Subscribe to our newsletter