Published

April 20, 2025

Axicabtagene ciloleucel, Axicabtagene ciloleucel [AXI01a, AXI01a]

Axicabtagene ciloleucel for treating relapsed/refractory diffuse large B-cell lymphoma (DLBCL), primary mediastinal cell lymphoma (PMBCL) and transformed lymphoma to DLBCL in patients previously treated with two or more lines of systemic therapy where the following criteria are met: This form is for the approval of leucapheresis and manufacture of CAR-T cells. There is a second part to this form which relates to the subsequent infusion of CAR-T cells and this will be available after submission of the first part. The second part of the form (AXI01b) can only be completed as a continuation of this first part of the form (AXI01a) and must be completed on infusion of CAR-T cells otherwise the treating Trust will not be reimbursed for the cost of axicabtagene ciloleucel, Axicabtagene ciloleucel for treating relapsed/refractory diffuse large B-cell lymphoma (DLBCL), primary mediastinal cell lymphoma (PMBCL) and transformed lymphoma to DLBCL in patients previously treated with two or more lines of systemic therapy where the following criteria are met: This form is for the approval of leucapheresis and manufacture of CAR-T cells. There is a second part to this form which relates to the subsequent infusion of CAR-T cells and this will be available after submission of the first part. The second part of the form (AXI01b) can only be completed as a continuation of this first part of the form (AXI01a) and must be completed on infusion of CAR-T cells otherwise the treating Trust will not be reimbursed for the cost of axicabtagene ciloleucel

  1. This application is being made by and that leucapheresis for and treatment with axicabtagene ciloleucel-modified CAR-T cells will be initiated by a consultant haematologist or medical oncologist specifically trained and accredited in the use of systemic anti-cancer therapy and working in an accredited CAR-T cell treatment centre and who is a member of the National CAR-T Clinical Panel for DLBCL, PMBCL and transformed lymphoma and a member of the treating Trust’s DLBCL, PMBCL and transformed lymphoma and CAR T cell multidisciplinary teams.
  2. The patient is either an adult (age 18 years or over) or a post-pubescent child (age <18 years) on the date of approval for axicabtagene ciloleucel by the National CAR-T Clinical Panel. Please mark below whether the patient is an adult or a post-pubescent child:
  • the patient is an adult OR
  • the patient is a post-pubescent child* *Please note that the use of axicabtagene ciloleucel is unlicensed in patients who are under 18 years old and so the Trust policy regarding the use of unlicensed medicines should be followed.
  1. The patient has a confirmed histological diagnosis of DLBCL or PMBCL or transformed lymphoma to DLBCL or post-transplant lymphoproliferative disorder or follicular lymphoma (FL) grade IIIB. Please tick appropriately below as to which type of lymphoma the patient has:
  • Diffuse large B-cell lymphoma (DLBCL) or
  • Primary mediastinal B-cell lymphoma (PMBCL) or
  • Transformed follicular lymphoma (TFL) to DLBCL or
  • Transformed marginal zone lymphoma (MZL) to DLBCL or -Transformed lymphoplasmacytoid lymphoma (LPL) to DLBCL or
  • Transformation of CLL to DLBCL (Richter’s transformation) or
  • Transformation of nodular lymphocyte predominant Hodgkin lymphoma (NLPHL) to DLBCL or
  • Post-transplant lymphoproliferative disease (PTLD) of DLBCL type and EBV positive or
  • Post-transplant lymphoproliferative disease (PTLD) of DLBCL type and EBV negative or
  • Follicular lymphoma grade 3B and as axicabtagene ciloleucel is unlicensed in this subtype of lymphoma, I also confirm that the Trust policy regarding the use of unlicensed medicines will be followed Note: Patients with Burkitt lymphoma or primary CNS lymphoma are not eligible for treatment with axicabtagene ciloleucel.
  1. The histological diagnosis of DLBCL or PMBCL or transformed lymphoma to DLBCL or post-transplant lymphoproliferative disorder of DLBCL type or FL grade 3B has been either made by or reviewed and confirmed by a designated lymphoma stem cell transplant centre.
  2. Prior to consideration of CAR-T cell therapy the patient’s disease has been re-biopsied unless either a biopsy at first relapse has shown DLBCL and there is progressive disease at second relapse at the same site or a biopsy is unsafe in which case the patient must have progressive disease at previously known sites of active disease. In such situations the original diagnostic biopsy review is acceptable. All patients with transformed lymphoma to DLBCL (TFL, MZL, CLL, NLPHL) or PTLD of DLBCL type or FL grade 3B who fulfil criteria 6 below must have a re-biopsy and have confirmation of DLBCL or FL grade 3B histology prior to consideration of CAR-T cell therapy. Please enter appropriately below as to which scenario applies to this patient: B-
  • re-biopsy at first relapse confirmed DLBCL or PMBCL and the patient has progressive disease at the same site or
  • re-biopsy at second relapse has confirmed DLBL or PMBCL or
  • re-biopsy at first or second relapse was/is unsafe plus there is progressive disease at previously documented sites of active disease and the previous histology was DLBCL or PMBCL or
  • re-biopsy at second relapse has again confirmed transformed lymphoma (TFL, MZL, CLL, NLPHL) to DLBCL or
  • re-biopsy at second relapse has again confirmed PTLD of DLBCL type or
  • re-biopsy at second relapse has again confirmed FL grade 3B
  1. The patient fulfils one of the following clinical scenarios relating to the definition of relapsed or refractory lymphoma and also the need for the patient to have received at least 2 previous lines of systemic therapy: please tick the appropriate box below. Refractory disease is defined as either progressive disease as the best response to the last line of systemic therapy or stable disease as the best response after at least 2 cycles of the last line of therapy with stable disease duration lasting no longer than 6 months from the last dose of the last line of systemic therapy. Relapsed disease is defined as disease that responded partially or completely to the last line of therapy and has since progressed. Progressive disease should be defined radiologically as per RECIST version 1.1 and be based on CT or MR scans and aided if necessary, after discussion at the National CAR T Clinical Panel, with the use of Lugano lymphoma response criteria. Second line treatment regimens which are appropriate include: R-GDP, R-GemOx, R-GemCarbo, R-ESHAP, R-ICE, R-IVE, R-BendaPola and the Marietta protocol. Neither radiotherapy nor steroids can be counted as a line of therapy. Please tick the box below which applies to this patient:
  • has DLBCL and received 2 or more lines of systemic therapy and relapsed after or was refractory to the last line of systemic therapy OR
  • had DLBCL with CNS involvement at first diagnosis and treated with first line systemic therapy (eg Marietta protocol) followed by stem cell transplantation as part of first line therapy and has relapsed after or was refractory to this first line of systemic therapy OR
  • has PMBCL and received 2 or more lines of systemic therapy and relapsed after or was refractory to the last line of systemic therapy OR
  • has transformed lymphoma to DLBCL (TFL, MZL, CLL, NLPHL) and received 2 or more lines of systemic therapy since diagnosis of transformation and relapsed after or was refractory to the last line of systemic therapy OR
  • has transformed lymphoma to DLBCL (TFL, MZL, NLPHL), received an anthracycline-containing regimen before transformation, and after transformation then received 1 or more lines of systemic therapy and relapsed after or was refractory to the last line of systemic therapy OR
  • has PTLD of DLBCL type and received 2 or more lines of systemic therapy since diagnosis of PTLD of DLBCL type and relapsed after or was refractory to the last line of systemic therapy OR
  • has FL grade 3B and received 2 or more lines of systemic therapy and relapsed after or was refractory to the last line of systemic therapy
  1. The patient has been previously treated with at least 3 cycles (2 cycles is allowed if there is outright refractory disease) of full dose anthracycline-containing regimen for his/her lymphoma or with the Marietta protocol if presenting with CNS involvement. Note: acceptable anthracycline-containing regimens include R-CHOP, Pola-R-CHP, R-CODOX-M/R-IVAC, DA-EPOC-R and the Marietta protocol.
  2. The patient has been previously treated with at least one anti-CD20 monoclonal antibody unless there is clear documentation of the determination of CD20 negative disease.
  3. Either the patient has not had stem cell transplantation (SCT) or has had an autologous or allogeneic SCT. Please tick one of the boxes below:
  • has not had SCT or
  • has had autologous SCT or
  • has had allogeneic SCT
  1. Either the patient has not previously been treated with an anti-CD19 antibody-drug conjugate or if previously treated with an anti-CD19 antibody-drug conjugate that a biopsy of the relapsed/refractory disease has been done and has been shown to be CD19 positive.
  2. Whether the patient has active CNS involvement by the lymphoma or not and if present whether this is in addition to systemic disease progression or not. Please tick one of the boxes below:
  • currently no known CNS involvement or
  • currently has both active CNS and systemic disease or
  • currently has isolated CNS disease only Note: patients with primary CNS lymphoma are not eligible for treatment with axicabtagene ciloleucel. Continued on the next page
  1. The patient has an ECOG performance score of 0 or 1. Please enter below as to the patient’s current ECOG performance status (PS): The ECOG performance status scale is as follows: PS 0 The patient is fully active and able to carry on all pre-disease performance without restriction PS 1 The patient is restricted in physically strenuous activity but is ambulatory and able to carry out work of a light or sedentary nature eg light house work, office work B- PS 2 The patient is ambulatory and capable of all selfcare but unable to carry out any work activities and is up and about more than 50% of waking hours PS 3 The patient is capable of only limited selfcare and is confined to bed or chair more than 50% of waking hours PS 4 The patient is completely disabled, cannot carry out any selfcare and is totally confined to bed or chair The patient currently has a performance status of either
  • ECOG PS 0 or
  • ECOG PS 1
  1. The patient has sufficient end organ function to tolerate treatment with CAR-T cell therapy.
  2. The patient has either had no previous therapy with any genetically modified autologous or allogeneic T cell immunotherapy or the patient has been treated with doses of genetically modified autologous or allogeneic T cell immunotherapy within an abandoned dosing cohort in a first in human dose-escalation phase I clinical trial. Please tick appropriate box as to which type of previous treatment the patient has had:
  • No previous therapy with any genetically modified autologous or allogeneic T cell immunotherapy or
  • Previously treated with doses of genetically modified autologous or allogeneic T cell immunotherapy within an abandoned dosing cohort in a first in human dose-escalation phase I clinical trial
  1. Prior to infusion 2 doses of tocilizumab are available for use in this patient in the event of the development of cytokine release syndrome.
  2. Axicabtagene ciloleucel-modified CAR-T cell therapy will otherwise be used as set out in its Summary of Product Characteristics (SPC).
  3. Approval for the use of axicabtagene ciloleucel has been formally given by the National DLBCL/PMBCL/TFL CAR-T cell Clinical Panel. Please state date of approval (DD/MM/YYYY)
  4. Following national approval for use of axicabtagene ciloleucel there has been local CAR-T cell multidisciplinary team agreement that this patient continues to have the necessary fitness for treatment and fulfils all of the treatment criteria listed here.

NHS funded From: 29 May 2023

Form version:

CDF Managed Access: NA

NICE Technology Appraisal: TA872 (28 February 2023)

Current Form Version

Note

The data on this page was produced using version 1.359 of the CDF list, downloaded from an archive of NHS England’s website on 11 April 2025 at 09:00.

If NHS England has published a new version of the CDF List but this site has not yet accessed that, this form may be out of date. Additionally, if any update has occurred without NHS England noting it as a change, this page will be out of date.