B:16.75”
T:16.5”
S:15.25”
CLINICAL NEWS
cell (RBC) transfusions. After treatment, of the seven subjects followed for six months
or longer, β-globin and HbAT87Q expression was sufficient for transfusion independence
in all four patients with a non-β0/β0 genotype. These patients remained transfusion-free
for at least 90 days (median = 287 days; range = 171-396 days) and, at the time of last
follow-up, their total Hb ranged from 9.1 to 12.2 g/dL.
Two patients with β0/β0 genotype have received a single RBC transfusion post-discharge,
the authors noted, while one patient with β0/β0 genotype remains transfusion-dependent.
After a median follow-up of 198 days post-infusion, the researchers found that the
safety profile was consistent with AHCT, with no grade 3 or higher treatment-related
adverse events recorded. There was also no clonal dominance or replication-competent
lentivirus observed.
“The updated data from the Northstar Study continue to show acceptable patient
safety with regard to myeloablation, drug product infusion risks, and genotoxicity at
this early follow-up,” said Dr. Walters, but he noted that “longer follow-up is needed to
understand the clinical significance of this data.” ●
REFERENCE
Walters MC, Rasko J, Hongeng S, et al. Update of results from the Northstar Study (HGB-204): a phase 1/2 study of gene therapy for beta-thalassemia
major via transplantation of autologous hematopoietic stem cells transduced ex-vivo with a lentiviral beta AT87Q-globin vector (LentiGlobin BB305
Drug Product). Abstract #201. Presented at the ASH Annual Meeting, December 6, 2015; Orlando, Florida.
His life. His treatment.
Proven joint bleed reduction in previously treated
adolescents and adults (ages 15 to 50) with
prophylaxis vs on demand1,3
Median number of joint bleeds per year1,3
On-demand (n=42)
Prophylaxis (n=42)
62% (26 out of 42)
Joint bleeds
per year
Range: 0-80.1
Joint bleeds
per year
Range: 0-18.4
of prophylaxis patients
experienced 0 joint bleeds
per year.3
Joint bleed results were not analyzed for significance.
Adults infusing with Kogenate FS prophylactically experienced an average of 2 joint bleeds annually,
compared with those infusing on demand, who experienced 29 joint bleeds annually.1,2
Dosing
Patients were randomized 1:1; all received Kogenate FS.
Prophylaxis regimen: 42 patients were to receive Kogenate FS 25 IU/kg three times per week. Escalation by 5 IU/kg (to 30 or
25 IU/kg at years 1 and 2, respectively) was allowed.
On-demand regimen: 42 patients were to receive Kogenate FS.
Primary endpoint: Bleeding frequency after all participants had completed at least 1 year of treatment.
P