12th Congress of the European Hematology ... - Haematologica

12 th Congress of the European Hematology Association
pts signed an informed consent and received nilotinib for an unapproved
indication. Results. Data are available on the 120 enrolled pts, of whom
97 (80.8%) pts were resistant and 22 (18.3%) were intolerant to imatinib.
67 (55.8%) were male. The median age was 58 (22-79) years and the
median time since first diagnosis of CML was 71.3 (2.2-298.2) mos. The
median duration of nilotinib exposure was 137.5 (2-503) days and median
average dose intensity for all pts was 796.6 (145.4-1149) mg/day.
Nilotinib dose was escalated for 26 (21.7%) pts. Treatment is ongoing
for 64 (53.3%) patients, and 56 (46.7%) have discontinued (25 [20.8%]
for disease progression, 14 [11.7%] for adverse events). Baseline mutation
data are available for 46 of the 64 pts. Of the 96 pts who had ≥6
months of follow-up, confirmed hematologic response (HR) occurred in
43 (44.8%) patients. 18 (18.8%) pts had complete HR, 9 (9.4%) had marrow
responses (no evidence of leukemia), and 16 (16.7%) returned to
chronic phase. Time to confirmed HR was 1 (0.8-3) month. Major CyR
occurred in 29 (30.2%) pts, of whom 15 (15.6%) had complete CyR and
14 (14.6%) had partial CyR. 12 (12.5%) pts had minor CyR, and 20
(20.8%) had minimal CyR. The rate of MCyR for resistant and intolerant
AP patients was 30.4% and 29.4%, respectively. Of the 120 patients
included in the safety analysis, overall the most frequent grade 3/4
adverse events included thrombocytopenia (n=41, 34.2%), neutropenia
(n=24, 20%), anemia (n=16, 13.3%), and elevated serum lipase (n=11,
9.2%). No pts experienced QTcF intervals >500 msec. Overall, there
were 9 deaths including 4 for disease progression, 2 due to other malignancies,
1 related to progressive disease complicated by a cerebral hemorrhage,
1 cardiac failure, and 1 due to sepsis. Summary and Conclusions.
These data demonstrated that nilotinib is clinically active and has an
acceptable safety and tolerability profile when administered to pts with
imatinib-resistant or -intolerant Ph + CML-AP.
0558
REDUCED INTENSITY CONDITIONING OF ALLO-SCT IN CML PATIENTS IS SUPERIOR
OVER MYELOABLATIVE CONDITIONING AND RESULTS WITH 60% OF LONG TERM
SURVIVAL
A. Lange, 1,2 M. Sedzimirska, 2 K. Suchnicki, 1 D. Duda, 2 E. Nowak, 2
S. Madej, 2 J. Lange2 1 L.Hirszfeld Inst. of Immunol. Exp. Ther., WROCLAW; 2 Lower Silesian Center
for Cellular Trans, WROCLAW, Poland
Background. In the era of Imatinib a number of allo SCT in CML
patients has been considerably reduced. However, in some patients we
come to the decision of allo SCT while toxicity or progression to more
advanced stages of the disease become clinically apparent. There are
rather rare events but still this problem may be relevant up to 20% of
patients especially during the first three years of treatment. An optional
proposal to proceed with allo SCT should have a rational basis behind.
Methods. In this study we present the data of allo SCT performed in one
center in two cohorts composed of patients receiving myeloablative
(cohort 1: Busulfan 16 mg/kg b.w., Cyclophosphamide 120 mg/kg b.w.
+ anti-thymocyte globulin - ATG 20 mg/kg b.w. in alternative donors
transplantation) and non myeloablative (cohort 2: Bu 8 mg/kg b.w., Flu
120 mg/kg, ATG usually 10 mg/kg b.w.) preparative regimens. Patients
characteristics in cohort 1 and cohort 2 are as follow: n=40 (n=58), 1st
CP: 31 (49), > 1st CP: 2 (9), ACCP or BC: 7 (0), sib/alternative donors:
30/10 (23/35), marrow/PBPC: 35/5 (14/44), years of transplant: 1989-
2000 (1999-2006). The results of the transplant procedure when compared
cohort 1 vs cohort 2 differed with respect to the incidence of transplant
related toxicity ≥ III grade (10/40 vs 1/58, p