Indication for treatment: Who is eligible to receive CAR T cells?

Author:  Julia Dobke, Editor:  Maria Yiallouros, English Translation:  Dr. med. Gesche Riabowol (geb. Tallen), Last modification: 2025/10/20 https://kinderkrebsinfo.de/doi/e217386

Treatment with CAR T cells in children and adolescents is currently limited to certain patients.

Patients with multiple recurrence‎s of acute lymphoblastic leukaemia‎ (ALL) or with ALL that does not respond to standard treatment (refractory‎ ALL) are eligible to receive CAR T-cell therapy.

One major criterion for the first group of above-mentioned paediatric patients to receive CAR T-cell therapy is that they have experienced multiple recurrences. Patients with a first recurrence of ALL usually receive a therapy combination of chemotherapy‎ (cytostatics‎) and, if necessary, monoclonal antibodies‎ as well as (if indicated) haematopoietic stem cell transplantation‎. CAR T-cell therapy is only considered if this therapy strategy is not effective, i.e. if a (complete) remission‎ has not been achieved or a new recurrence occurs.

The efficacy of CAR T-cell therapy as well as how the patients are tolerating the treatment (tolerability) is also being evaluated in clinical trials for children and adolescents with CD19-positive lymphoma‎.

Clinical requirements for eligibility of CAR T-cell therapy

Patients who are eligible for CAR T-cell therapy must meet certain clinical re-quirements. These include:

  • that the patient has sufficient T lymphocytes‎ of his own,
  • that the patient's leukaemia cells present the correct antigen‎ (CD19) on their cell wall so that the CAR T cells can "dock" there,
  • that the time interval from a previous stem cell transplantation‎ is at least 100 days,
  • that there were no rejection reactions during the pre-treatment (donor-versus-recipient reaction, also called graft-versus-host disease‎, GvHD),
  • that the patient is in stable clinical condition without severe organ damage.

Personalised treatment

Treatment with CAR T cells as a personalised treatment for children and adolescents with acute lymphoblastic leukaemia (ALL) is possible in Europe with an approved product from Novartis (tisagenlecleucel - Kymriah®).

Study participation

Other CAR T-cell therapies are currently being assessed for their safety and efficacy in early clinical trials, so-called phase I and II studies. If CAR T-cell therapy is part of a study, patients will be "screened" to see whether they meet the inclusion criteria for participation in the study. Aside from health aspects, these may also include the type of cancer and the age of the patient. If a patient turns out to be eligible to participate, informed consent forms must be signed by the patient’s custodians as well as assents by the patient (depending on their age).