spinal muscular atrophy, severe combined immunodeficiency due to adenosindesamniase deficiency, metachromatic leukodystrophy, folicular lymphoma, mantle cell lymphoma, diffuse large B-cell lymphoma, other and unspecified types of non-Hodgkin's lymphoma
Conditions
Interventions
Group 1: Patients who receive ATMP treatment with a gene therapy approved in Germany in connection with one of the four diseases spinal muscular atrophy, severe combined immunodeficiency due to adenos
Sponsors
Universitätsklinikum Heidelberg, Klinik für Hämatologie, Onkologie und Rheumatologie Innere Medizin V
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Participating patients must be insured with a statutory health insurance - Signed treatment contract between practitioner and patient/parents according to §630a BGB - Treatment in one of the participating treatment centers - Age of CAR-T-cell therapy patients: = 18 years - Age of patients for SMA, ADA-SCID and MLD: ? 7 years - Ability to understand the patient's information and sign the consent form - Ability to consent by patients themselves or by parents - Consent to the exchange of data between practitioners, the telemedicine platform and the INTEGRATE register - Indication for ATMP treatment within the existing marketing authorisation for one of the following indications prior to inclusion: o CAR-T cell therapy: patients with diseases of the lymphatic system and planned commercial CAR-T cell therapy in an approved indication o Spinal muscular atrophy (SMA): patients with SMA and planned commercial gene therapy with onasemnogen abeparvovec in the approved indication o Severe combined immunodeficiency in adenosine desaminase deficiency (ADA-SCID): patients with ADA-SCID and planned gene therapy with autologous CD34+ cells transduced to express adenosine desaminase (ADA) in the approved indication. o Metachromatic leukodystrophy (MLD): patients with MLD and planned gene therapy in the approved indication (Atidarsagen Autotemcel – autologous lentiviral transduced CD34+ cells to replace the arylsulfatase A gene).
Exclusion criteria
Exclusion criteria: - ATMP patients without a statutory health insurance - Treatment with ATMP as part of a clinical trial or off-label use - patients that are not able to give informed consent - Project content is not comprehensible to patients
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Health-related quality of life of patients | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Patient and healthcare provider satisfaction with care. 2) Overall survival 3) Event-free survival including adverse drug reaction (ADR) recording & disease-specific endpoints: Lymphoma: progression, recurrence of the underlying disease, death SMA: free sitting >30 seconds MLD: gross motor function ADA-SCID: Immune reconstitution | — |
Countries
Germany
Contacts
Public ContactClaudia Ganter
Universitätsklinikum Heidelberg
Outcome results
None listed