Pharmacoeconomics in the Application of 5-azacitidine in the Treatment of Myelodysplastic Syndromes and Acute Myeloid Leukemia

The investigators want to compare the global response rate of patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) after six months of treatment with 5-azacitidine on two different doses. First group of 50 mg/m2 for 10 days each 28 days versus 75 mg/m2 for 7 days on 28 days cycles.

Study Overview

• Status: Withdrawn
• Conditions: Acute Myeloid Leukemia; Myelodysplastic Syndromes
• Intervention / Treatment: Drug: 5-azacitidine

Detailed Description

5-Azacitidine it's the standard treatment for patients with MDS and it is widely used for AML in patients that aren't fit for intensive treatment. The standard dose of azacitidine is 75 mg/m2 for 7 días on 28 days cycles. Nevertheless there are studies of pharmacokinetics that have showed there is no difference on plasmatic distribution between 25, 50, 75 or 100 mg/m2 with similar concentration curves. The actual presentation of azacitidine is 100 mg per bottle with less of 24 hours of utility after opened. Looking for optimizations on the use of 5-azacitidine, we have changed the dosing to 50 mg/m2 for 10 days on 28 days cycles with a total dose application of 95% of the traditional regimen with the saving of 5 bottles of the drug.

• Study Type: Observational

Contacts and Locations

Study Locations

• Mexico
  • Estado De México
    • Huixquilucan, Estado De México, Mexico, 52763
      • Grupo Cooperativo de Hemopatías Malignas

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

1. Confirmed diagnosis of MDS that require treatment with 5-aza characterized by: i) RAEB-1 defined as the presence of 5%-9% of myeloblasts in bone marrow; ii) RAEB-2 defined as the presence of 10%-19% of myeloblasts in bone marrow or 5%-19% blasts in peripheral blood; iii) IPSS-R very high, high or intermediate
2. Confirmed AML with blasts ≥ 20% in bone marrow or peripheral blood and that its not candidate to receive intense chemotherapy at consideration of the physician.

Description

Inclusion Criteria:

• Age ≥18 years
• Confirmed diagnosis of MDS that require treatment with 5-aza characterized by: i) RAEB-1 defined as the presence of 5%-9% of myeloblasts in bone marrow; ii) RAEB-2 defined as the presence of 10%-19% of myeloblasts in bone marrow or 5%-19% blasts in peripheral blood; iii) IPSS-R very high, high or intermediate
• Confirmed AML with blasts ≥ 20% in bone marrow or peripheral blood and that its not candidate to receive intense chemotherapy at consideration of the physician.
• Performance status of 0, 1 o 2 by the Eastern Cooperative Oncology Group (ECOG).
• Availability to sign an informed consent

Exclusion Criteria:

• Previous treatment for MDS or AML with chemotherapy or another antineoplastics including hypomethylating agents.
• Acute promyelocytic leukemia

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
A; Azacitidine 50 mg/m2 for 10 days each 28 days	Drug: 5-azacitidine; Cycles of 28 days
B; Azacitidine 75 mg/m2 for 7 days each 28 days	Drug: 5-azacitidine; Cycles of 28 days

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Response Rate	Global response rate by International Criteria	6 months

Collaborators and Investigators

• Sponsor: Grupo Cooperativo de Hemopatías Malignas

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2020
• Primary Completion (Anticipated): December 31, 2021
• Study Completion (Anticipated): March 15, 2022

Study Registration Dates

• First Submitted: March 3, 2020
• First Submitted That Met QC Criteria: March 3, 2020
• First Posted (Actual): March 5, 2020

Study Record Updates

• Last Update Posted (Actual): July 9, 2021
• Last Update Submitted That Met QC Criteria: July 5, 2021
• Last Verified: July 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Histologic Type; Neoplasms; Disease; Bone Marrow Diseases; Hematologic Diseases; Precancerous Conditions; Syndrome; Myelodysplastic Syndromes; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Preleukemia; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antimetabolites, Antineoplastic; Antimetabolites; Antineoplastic Agents; Azacitidine
• Other Study ID Numbers: HAL 318/2019

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No