An observational, Post-Authorisation Safety Study (PASS) to describe the safety and effectiveness of tabelecleucel in patients with Epstein-Barr Virus positive (EBV+) Post-Transplant Lymphoproliferative Disease (PTLD) in a real-world setting in Europe: EBVOLVE study

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  EBVALLO
• Study drug International non-proprietary name (INN) or common name:
  TABELECLEUCEL
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XL09) tabelecleucel

  tabelecleucel
• Additional medical condition(s):
  Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease (EBV+PTLD)

Population studied

• Short description of the study population:
  adult and paediatric patients 2 years of age and older with relapsed or refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate
• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adult and elderly population (≥18 years)

  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Elderly (≥ 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)
• Special population of interest:
  Nursing women

  Other

  Pregnant women
• Special population of interest, other:
  patients with organ impairment

Study design details

• Study design:
  This study is an observational, multicenter, multinational PASS with the primary objective of further characterising the safety profile of tabelecleucel and with secondary objectives including the real-world effectiveness of tabelecleucel in patients with EBV+ PTLD following HCT or SOT.
• Main study objective:
  Primary objective : To describe the safety of tabelecleucel in patients with EBV+ PTLD following hematopoietic cell transplantation (HCT) or solid organ transplantation (SOT) in a real-world setting. ; Secondary objectives: ; 1. To describe the effectiveness of tabelecleucel in patients with EBV+ PTLD following HCT or SOT in a real-world setting. ; 2. To describe the patient population treated with tabelecleucel for EBV+ PTLD following HCT or SOT in a real-world setting. ; 3. To describe tabelecleucel treatment patterns, including dosing and schedule, in patients with EBV+ PTLD following HCT or SOT in a real-world setting.
• Setting:
  The study will be conducted in the real-world setting in institutions experienced in the treatment of cancer and having the permission for allogeneic cell manipulation. ; Countries/Region: The study will be conducted in all European countries where tabelecleucel is commercially available in accordance with the launch sequence for commercialisation, which will depend on country-specific reimbursement timelines. There is no limit to the number of patients to be enrolled in the study.; Study Sites: All physicians who are treating one or more patients with tabelecleucel will ; be asked to participate in this study, and patients will be asked for consent.; The PASS population will consist of EBV+ PTLD patients who are prescribed tabelecleucel in the real-world setting in Europe and who consent for the secondary use of existing data collected for the purpose of providing tabelecleucel (available in the HCP portal database). In addition, patients will be asked to consent for additional primary data collection in the PASS eCRF system. These data will be obtained on routine clinical practice visits, not collected in the HCP portal database but required to meet all the study objectives.
• Outcomes:
  Safety, effectiveness, demographics, baseline and clinical characteristics, tabelecleucel treatment information
• Data analysis plan:
  For all subjects enrolled in the PASS, subject-level data will be available for the purposes of analysis and annual reporting. ; ; Statistical analyses will be fully described in a Statistical Analysis Plan (SAP). Analyses will be performed on an annual basis. ; Analyses will be descriptive in nature, as no hypothesis will be tested. In general, missing data will not be imputed (except for dates) and the data will be analysed according to the complete case approach. The frequency of missing data will be indicated in descriptive tables for all variables.; Summary data will be provided for all variables collected and the data will be reported for the whole population and by subgroups.