Trial Outcomes & Findings for Vatalanib in Treating Patients With Primary or Secondary Myelodysplastic Syndromes (NCT NCT00072475)

A total of 155 participants were enrolled between December 2003 and April 2008.

Of the 155 participants recruited, 2 participants cancelled prior to starting treatment; 7 were determined to have AML at registration and 4 had diagnosis other than MDS. Thus 142 participants were evaluable for response and 153 for adverse events.

Vatalanib in Treating Patients With Primary or Secondary Myelodysplastic Syndromes

Response was measured by International Standardized Response Criteria for MDS * Complete Response: Bone marrow showing \< 5% myeloblasts with normal maturation of all cell lines; Hgb \> 11 g/dL (untransfused), ANC ≥1.5 K/L, PLT ≥ 100 K/L, No blasts, no dysplasia * Partial remission: All of the CR criteria (if abnormal at baseline), except BM evaluation. Blasts decreased by ≥ 50% over baseline. Cellularity and morphology are not relevant. Hematologic improvement: * Erythroid (HI-E): For participants with baseline HGB \< 11g/dL, Major: \> 2g/dL increase, transfusion independence. Minor: 1-2g/dL increase, ≥ 50% decrease in transfusion requirements * Platelet (HI-P): For participants with baseline PLT \< 100 K/L: Major: absolute increase of \> 30 K/L, transfusion independence. Minor: ≥ 50% increase (net increase of \>10 K/L) * Neutrophil (HI-N): For participants with baseline ANC \< 1.5 K/L, Major: \> 100% increase (net increase \> 0.5 K/L). Minor: \> 100% increase (absolute increase \< 0.5 K/L)

Time to transformation to AML is defined as the time from registration to the transformation of MDS to AML or death of any cause. Participants not meeting these criteria were censored at the date of last follow-up. This outcome was estimated using the Kaplan Meier method.

Duration of response (DOR) was defined as the time from response (complete remission, partial remission or hematologic improvement) to progression or death of any cause. Responding and alive patients were censored at the date of last follow-up. The median DOR with 95% CI was estimated using the Kaplan Meier method. Response was measured by International Standardized Response Criteria for MDS (described in above outcome measure).

Overall survival (OS) as the interval from the on-study date until death. OS was estimated using the Kaplan Meier method.

Progression free survival (PFS) was defined as the time from registration to progression or death of any cause. Progression free and alive patients were censored at the date of last clinical assessment. The median PFS with 95% CI was estimated using the Kaplan Meier method. Progression is defined as * For patients with \<5% bone marrow blasts: ≥50% increase in blasts to \>5% blasts * For patients with 5-10% bone marrow blasts: ≥50% increase to \>10% blasts * For patients with 10-19% bone marrow blasts: increase to ≥20% blasts * One or more of the following: 50% or greater decrement from maximum remission/response levels in ANC \< 1.5 K/L or PLT\< 100 K/L, or reduction in HGB by at least 2 g/dL or becoming transfusion dependent Progression after HI: Includes one or more of the following * Decrement of 50% or greater from maximum response levels in ANC \< 1.5 K/L or PLT \< 100 K/L * Reduction in HGB concentration by at least 2 g/dL * Becoming transfusion dependent