How LYFGENIA was studied
The safety of LYFGENIA was studied in individuals with sickle cell disease in 1 clinical trial and 1 long-term follow-up study.
The clinical trial
included:
54
people
who initiated stem cell collection†
Individuals with a
median age of
25
years
(min age 12 years, max age 43 years)
The median duration of
follow-up for individuals
treated with LYFGENIA†:
42
months
(min 12 months; max 87 months)
The clinical trial
included:
54
people
who initiated stem cell collection†
Individuals with a
median age of
25
years
(min age 12 years, max age 43 years)
The median duration of
follow-up for individuals
treated with LYFGENIA†:
42
months
(min 12 months; max 87 months)
†45 people were treated with LYFGENIA.
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LYFGENIA results Before and after LYFGENIA infusion More about the studyJump to topics on page:
LYFGENIA resultsBefore and after LYFGENIA infusion
More about the study
A one-time transformational gene therapy with the potential to decrease or stop vaso-occlusive events (VOEs)
A one-time transformational gene therapy with the potential to decrease or stop vaso-occlusive events (VOEs)
The efficacy of LYFGENIA was studied in 36 individuals. 32 individuals were evaluated for the number of
vaso-occlusive events (VOEs)vaso-occlusive events (VOEs)In a LYFGENIA clinical study, VOEs were defined as any of the following events requiring evaluation at a medical facility: an episode of acute pain with no medically determined cause other than vaso-occlusion, lasting more than 2 hours, acute chest syndrome, acute hepatic sequestration, or acute splenic sequestration they experienced between 6-18 months after treatment.
did not experience any vaso-occlusive events (VOEs)vaso-occlusive events (VOEs)In a LYFGENIA clinical study, VOEs were defined as any of the following events requiring evaluation at a medical facility: an episode of acute pain with no medically determined cause other than vaso-occlusion, lasting more than 2 hours, acute chest syndrome, acute hepatic sequestration, or acute splenic sequestration(28/32 individuals)
did not experience any vaso-occlusive events (VOEs)vaso-occlusive events (VOEs)In a LYFGENIA clinical study, VOEs were defined as any of the following events requiring evaluation at a medical facility: an episode of acute pain with no medically determined cause other than vaso-occlusion, lasting more than 2 hours, acute chest syndrome, acute hepatic sequestration, or acute splenic sequestration(28/32 individuals)
did not experience any severe vaso-occlusive events (sVOEs)severe vaso-occlusive events (sVOEs)In a LYFGENIA clinical study, sVOEs were defined as vaso-occlusive events (VOEs) requiring a hospitalization or multiple visits to an emergency department/urgent care over 72 hours and receiving IV medications at each visit, or priapism requiring any level of medical attention(30/32 individuals)
did not experience any severe vaso-occlusive events (sVOEs)severe vaso-occlusive events (sVOEs)In a LYFGENIA clinical study, sVOEs were defined as vaso-occlusive events (VOEs) requiring a hospitalization or multiple visits to an emergency department/urgent care over 72 hours and receiving IV medications at each visit, or priapism requiring any level of medical attention(30/32 individuals)
What else should I know about the study?
4 individuals experienced vaso-occlusive events (VOEs) after 18 months. These individuals did not experience any VOEs
between 6-18 months after treatment
No clinically meaningful differences in efficacy and safety were observed between adults and adolescents
38 months was the median duration of follow-up for efficacy for the 36 individuals who received LYFGENIA
(min of 12 months; max of 61 months)
5 individuals with a history of stroke or vasculopathy were treated. They were at least 18 years old and on chronic
transfusion therapy prior to LYFGENIA infusion. At 44-60 months’ follow-up, all individuals remain transfusion independent
without repeated stroke
Vaso-occlusive events (VOEs) before and after LYFGENIA infusion
sVOEs were also counted as VOEs.
This figure is not included in the LYFGENIA Prescribing Information.
What are vaso-occlusive events (VOEs)?
What are vaso-occlusive events (VOEs)?
In the LYFGENIA clinical study, vaso-occlusive events (VOEs) were defined as any of the following events, which need to be evaluated at a medical facility:
- an episode of acute pain with no medically determined cause other than vaso-occlusion, lasting more than 2 hours
- acute chest syndrome
- acute hepatic sequestration
- acute splenic sequestration
What are severe vaso-occlusive events (sVOEs)?
What are severe vaso-occlusive events (sVOEs)?
In the LYFGENIA clinical study, severe vaso-occlusive events (sVOEs) were defined as either of the following events:
- VOE requiring a hospitalization or multiple visits to an emergency department/urgent care over 72 hours and receiving intravenous medications at each visit
- priapism requiring any level of medical attention