Dataset

Sargramostim N=52^* Median (25%, 75%)

Placebo N=47 Median (25%, 75%)

p-value^** 

ANC>500mm^{3 a}

13 (11, 16)

17 (13, 25)

0.009

ANC>1000mm^{3 b}

14 (12, 18)

21 (13, 34)

0.003

PLT>20,000mm^{3 c}

11 (7, 14)

12 (9, >42)

0.10

RBC^d

12 (9, 24)

14 (9, 42)

0.53

^* Patients with missing data censored.
^a 2 patients on Sargramostim and 4 patients on placebo had missing values.
^b 2 patients on Sargramostim and 3 patients on placebo had missing values.
^c 4 patients on placebo had missing values.
^d 3 patients on Sargramostim and 4 patients on placebo had missing values.
^** p=Generalized Wilcoxon

The incidence of severe infections and deaths associated with infections was significantly reduced in patients who received Leukine. During induction or consolidation, 27 of 52 patients receiving Leukine and 35 of 47 patients receiving placebo had at least one grade 3, 4 or 5 infection (p=0.02). Twenty-five patients receiving Leukine and 30 patients receiving placebo experienced severe and fatal infections during induction only. There were significantly fewer deaths from infectious causes in the Sargramostim arm (3 versus 11, p=0.02). The majority of deaths in the placebo group were associated with fungal infections with pneumonia as the primary infection.

Disease outcomes were not adversely affected by the use of Leukine. The proportion of patients achieving complete remission (CR) was higher in the Leukine group (69% as compared to 55% for the placebo group), but the difference was not significant (p=0.21). There was no significant difference in relapse rates; 12 of 36 patients who received Leukine and 5 of 26 patients who received placebo relapsed within 180 days of documented CR (p=0.26). The overall median survival was 378 days for patients receiving Leukine and 268 days for those on placebo (p=0.17). The study was not sized to assess the impact of Leukine treatment on response or survival. 

Mobilization and Engraftment of PBPC

A retrospective review was conducted of data from patients with cancer undergoing collection of peripheral blood progenitor cells (PBPC) at a single transplant center. Mobilization of PBPC and myeloid reconstitution post-transplant were compared between four groups of patients (n=196) receiving Leukine for mobilization and a historical control group who did not receive any mobilization treatment [progenitor cells collected by leukapheresis without mobilization (n=100)]. Sequential cohorts received Leukine. The cohorts differed by dose (125 or 250 mcg/m²/day), route (IV over 24 hours or SC) and use of Leukine post transplant . Leukaphereses were initiated for all mobilization groups after the WBC reached 10,000/mm³. Leukaphereses continued until both a minimum number of mononucleated cells (MNC) were collected (6.5 or 8.0 x 10⁸/kg body weight) and a minimum number of phereses (5-8) were performed. Both minimum requirements varied by treatment cohort and planned conditioning regimen. If subjects failed to reach a WBC of 10,000 cells/mm³ by day 5 another cytokine was substituted for Leukine; these subjects were all successfully leukapheresed and transplanted. The most marked mobilization and post transplant effects were seen in patients administered the higher dose of Leukine (250 mcg/m²) either IV (n=63) or SC (n=41).