VIDAZA® Treatment for MDSa

Clinical Trials

VIDAZA® is the standard of care for higher-risk MDSa patients1

VIDAZA® clinical trials have shown:

  • VIDAZA® offers patients
    • improved quality of life2
    • significantly greater treatment response vs. supportive care3
    • significantly delayed time to AML or death vs. supportive care3
  • Treatment should be continued as long as the patient continues to benefit or until disease progression.4
  • Continued treatment with VIDAZA® can increase response quality in responders4

VIDAZA® has been investigated in two phase III studies, AZA-0015 and CALGB 9221.3

AZA-001 Clinical Study

AZA-001: 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.1

Primary objective

Overall survival. All patients followed until death or study closure

Secondary objective

  • Time to AML or death, haematological response and improvement
  • RBC transfusion independence, infections requiring IV therapy and adverse events

Inclusion criteria

Patients were eligible for enrolment if they were aged ≥ 18 years, with Int-2 or high-risk MDS and RAEB, RAEB-T, or CMML with at least 10% bone marrow blasts with a white blood count below 13 x 109/L1

Randomisation of Clinical Trial Participants

  • Best supportive care was included in each arm
  • Treatment continued until unacceptable toxicity, transformation to AML, or disease progression occurred
  • No erythropoiesis stimulating agents were allowed1

Patient characteristics1

  • 358 patients were randomised at 79 sites
    • VIDAZA® (n=179) or CCR (n=179): BSC only (n=105, 59%), LDAC (n=49, 27%), or Intensive CT (n=25, 14%)
  • Median age: 69 years (range 38-88 years)
  • The VIDAZA® and CCR groups were comparable for baseline (BL) parameters
    • at BL, 95% of patients were higher-risk: RAEB (58%), RAEB-T/WHO AML (34%), CMML (3%), and other (5%).
    • by IPSS, 89% were higher-risk: Int-2 (43%), High (46%), and 11% indeterminate/other
  • VIDAZA® was administered for a median of nine cycles; LDAC for four cycles
  • Follow-up period of 21.1 months

Results

  • VIDAZA® doubles the 2-year survival rate from 26% to 51% (p<0.0001) compared with conventional care regimens (CCR)
  • VIDAZA® treatment was associated with a median overall survival of 24.5 months vs. 15 months for those receiving CCR
  • The survival benefits of VIDAZA® were consistent regardless of CCR treatment option used in the control arm
  • VIDAZA® led to a significantly reduced need for red blood cell (RBC) transfusions compared to CCR
  • VIDAZA® delayed time to AML progression or death in Int-2 and High-risk MDS patients compared to CCR (17.8 months vs. 11.5 months, respectively; p<0.0001)
  1. Fenaux P et al. Lancet Oncol 2009; 10:223-32.

CALGB 9221 Clinical Study

CALGB 9221: Randomized Controlled Trial of Azacitidine in Patients With the Myelodysplastic Syndrome: A Study of the Cancer and Leukemia Group B1

Primary objective

Overall response (sum of complete response and partial response)

Secondary objectives1

  • Overall survival
  • Time to AML transformation or death
  • Transfusion independence

Inclusion criteria2

Patients with refractory anaemia (RA) or RA with ringed sideroblasts (RARS) met additional criteria of significant bone marrow dysfunction.

Design1

  • Phase III, randomised, controlled trial
  • Both arms received transfusions and antibiotics as required
  • Patients in supportive care arm (whose disease worsened) were permitted to cross over to VIDAZA® after at least 4 months of supportive care
  • Use of all haematopoietic growth factors was prohibited

Randomisation

Adapted from Silverman LR et al. J Clin Oncol 2002; 20:2429-2440.

Patient characteristics1

  • Median age: 68 years
  • Males (69%)
  • The two arms were evenly balanced at entry

Results

  • Of the VIDAZA®-treated patients, 7% had a complete response, 16% had a partial response and 37% showed haematological improvement
  • Transformation to AML (as a first event) occurred in 15% of patients with VIDAZA® vs. 38% on supportive care (P=0.001)
  • Of 65 patients with RBC transfusion dependence at baseline, 45% became transfusion-independent
  • The VIDAZA® arm had an increase in the number of RBC transfusions in the first month of treatment, but decreased subsequently
  • A landmark analysis conducted to compare survival at six months demonstrated significantly higher survival with VIDAZA® vs. supportive care (18 months vs. 11 months respectively, p=0.03)
  • QoL assessments found significant advantages in physical function, symptoms and psychological state for patients who crossed over to VIDAZA® as measured according to the European Organization for Research and Treatment of Cancer (EORTC) and the Mental Health Inventory (MHI)1
  1. Silverman LR et al. J Clin Oncol 2002; 20: 2429–2440.
  1. Fenaux P. ASH 2007; Abstract 817.
  2. Kornblith AB et al. J Clin Oncol. 2002. 20: 2441-2452.
  3. Silverman LR et al. J Clin Oncol. 2002. 20: 2429–2440.
  4. Silverman LR et al. Cancer. 2011. 117: 2697–2702.
  5. Fenaux P et al. Lancet Oncol 2009; 10:223-32.
a. MDS-specific Indication

VIDAZA® is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation with:

  • intermediate-2 and high-risk myelodysplastic syndromes (MDS) according to the International Prognostic Scoring System (IPSS),
  • chronic myelomonocytic leukaemia (CMML) with 10-29% marrow blasts without myeloproliferative disorder
b. AML-specific Indication:

Treatment of adult AML patients who are not eligible for haematopoietic stem cell transplantation with 20-30% blasts and multi-lineage dysplasia, according to the WHO classification. WHO classifies RAEB-T patients with blasts ≥20% to <30% as AML patients.

Disclaimer

This is an international website for VIDAZA® and is intended for healthcare professionals outside the US. The information on this site is not country-specific and may contain information that is outside the approved indications in the country in which you are located.

The information on this website is based on the European Summary of Product Characteristics (SmPC). Please refer to your country-specific website, or contact a Celgene representative in your country for the latest information specific to your country.