title: Immunotherapy with the trifunctional anti-CD20 x anti-CD3 antibody FBTA05 (Lymphomun) in paediatric high-risk patients with recurrent CD20-positive B cell malignancies.
abstract: Children with B cell malignancies refractory to standard therapy are known to have a poor prognosis and very limited treatment options. Here, we report on the treatment and follow-up of ten patients diagnosed with relapsed or refractory mature B-cell Non Hodgkin Lymphoma (B-NHL), Burkitt leukaemia (B-AL) or pre B-acute lymphoblastic leukaemia (pre B-ALL). All children were treated with FBTA05 (now designated Lymphomun), an anti-CD3 x anti-CD20 trifunctional bispecific antibody (trAb) in compassionate use. Within individual treatment schedules, Lymphomun was applied (a) after allogeneic stem cell transplantation (allo-SCT, n = 6) to induce sustained long-term remission, or (b) stand alone prior to subsequent chemotherapy to eradicate residual disease before allo-SCT (n = 4). Nine of ten children displayed a clinical response: three stable diseases (SD), one partial remission (PR) and five induced or sustained complete remissions (CR). Five of these nine responders died during follow-up. The other patients still maintain CR with a current overall survival of 874-1424 days (median: 1150 days). In conclusion, despite the dismal clinical prognosis of children refractory to standard therapy, immunotherapy with Lymphomun resulted in a favourable clinical outcome in this cohort of refractory paediatric patients.
authors.affiliation: Department of Paediatric Oncology, Haematology and Immunology, University of Duesseldorf, Duesseldorf, Germany.
authors.forename: Friedhelm R
authors.initials: FR
authors.lastname: Schuster
authors.forename: Michael
authors.initials: M
authors.lastname: Stanglmaier
authors.forename: Wilhelm
authors.initials: W
authors.lastname: Woessmann
authors.forename: Beate
authors.initials: B
authors.lastname: Winkler
authors.forename: Meinolf
authors.initials: M
authors.lastname: Siepermann
authors.forename: Roland
authors.initials: R
authors.lastname: Meisel
authors.forename: Paul G
authors.initials: PG
authors.lastname: Schlegel
authors.forename: Jürgen
authors.initials: J
authors.lastname: Hess
authors.forename: Horst
authors.initials: H
authors.lastname: Lindhofer
authors.forename: Arndt
authors.initials: A
authors.lastname: Borkhardt
authors.forename: Raymund
authors.initials: R
authors.lastname: Buhmann
mesh_terms: D000293:Adolescent; D064591:Allografts; D018033:Antibodies, Bispecific / Q000008:administration & dosage* / Q000009:adverse effects; D002051:Burkitt Lymphoma / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007167:Immunotherapy; D008297:Male; D018365:Neoplasm, Residual; D015452:Precursor B-Cell Lymphoblastic Leukemia-Lymphoma / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D033581:Stem Cell Transplantation; D015996:Survival Rate
chemical_list: D018033:Antibodies, Bispecific; C530113:Bi20 antibody
keywords: B-cell malignancies; anti-CD20 x anti-CD3; bispecific antibody; children; immunotherapy