National implementation of the use of tisagenlecleucel in paediatric and young adult patients with acute lymphoblastic leukaemia (ALL) in National Health Service England (NHSE)
Furness, C. L. Hough, R. Cummins, M. Murphy, G. Ghorashian, S. Amrolia, P. Roddie, C. O'Reilly, M. Wynn, R. Bonney, D.
Furness, C. L.
Hough, R.
Cummins, M.
Murphy, G.
Ghorashian, S.
Amrolia, P.
Roddie, C.
O'Reilly, M.
Wynn, R.
Bonney, D.
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Abstract
Background: Following European Medicines Agency
(EMA) approval of Tisagenlecleucel (KYMRIAH®) for the
treatment of relapsed/refractory acute lymphoblastic leukaemia
(ALL) in children and young adults in 2018, England
established a structured funding programme via the
National Health Service England (NHSE) Cancer
Drugs Fund.
Methods: NHSE established a national CAR-T clinical
panel for ALL (NCCP ALL). The aim of the NCCP ALL
was to review cases to confirm eligibility against published
criteria in line with the ELIANA study inclusion criteria and
ensure prompt national access to Tisagenlecleucel. All
CAR-T centres (3 open at NCCP set up and 9 open at 1 year
review) were accredited for the Immune Effector Cell
Standards in the 7th edition of the JACIE Standards in line
with EBMT/JACIE recommendations (Yakoub-Agha et al
2019). The NCCP ALL consisted of representatives from
NHSE, CAR-T centres, patient representation and independent
ALL clinical experts. We describe panel experience
over a one year period.
Results: The NCCP met by weekly teleconference to
review cases referred by individual CAR-T centre lead
clinicians who endorsed eligibility. Approval for access to
Tisagenlecleucel was granted through unanimous panel consensus agreement following review of eligibility criteria
(according to disease and CD19+ status, absence of CNS
disease, performance status and organ function). Allocation
to centre was achieved by review of geography, slot
availability and patient preference.
From 19.11.2018-18.11.2019 34 patients were
approved (age range 9 months - 21 years, median age 10.5
years). 5 did not proceed to leukapheresis (2 incorrect
diagnosis of relapse, 1 received alternative CAR-T clinical
trial product, 2 unable to proceed due to disease
progression/complications). At time of abstract submission
23/29 (79.3%) patients had undergone CAR-T infusion.
3 patients who underwent leukapheresis were unable
to proceed to infusion (1 patient due to emergence of
CD19 negative disease, 1 patient due to CNS disease
progression, 1 patient received cranial radiotherapy to
control CNS disease and suffered frank bone marrow
relapse treated with Inotuzumab followed by a failed
manufacture). 3 patients await infusion. Mean time from
panel approval to leukapheresis was 15 days (range 0-47
days) and mean time from leukapheresis to CAR-T infusion
for patients infused was 64 days (range 35 - 92 days)
Of 15 patients evaluable beyond 100 days (8 patients yet
to reach this time point), 11 have a documented status of
‘alive in MRD negative remission’. This represents 73%
patients infused (11/15). Brief toxicity and follow up data
will be reported at EBMT 2020 with future planned efficacy
analysis.
Conclusions: The establishment of a national panel in
England for Tisagenlecleucel approval has allowed prompt,
equitable and trackable access to this CAR-T product for
ALL. From a worldwide perspective, NHSE is one of the
first health services to introduce a national co-ordinated
access programme of care and will utilise programme data
to assess real world outcomes for patients treated with
Tisagenlecleucel.
Affiliation
Description
Date
2020
Publisher
Collections
Keywords
Type
Meetings and Proceedings
Citation
Furness CL, Hough R, Cummins M, Murphy G, Ghorashian S, Amrolia P, et al. National implementation of the use of tisagenlecleucel in paediatric and young adult patients with acute lymphoblastic leukaemia (ALL) in National Health Service England (NHSE). Bone Marrow Transplantation. 2020;55(SUPPL 1):228-9.