Teclistamab, a B-cell maturation antigen (BCMA) × CD3 directed bispecific antibody, has shown high response rates and durable remissions in the MAJESTEC-1 trial in patients with relapsed and refractory multiple myeloma (RRMM). We retrospectively assessed efficacy and tolerability in 123 patients treated at 18 different German centers to determine whether outcome is comparable in the real-world setting. Most patients had triple-class (93%) or penta-drug (60%) refractory disease, 37% of patients had received BCMA-directed pretreatment including idecabtagene vicleucel (ide-cel) CAR-T cell therapy (21/123, 17.1%). With a follow-up of 5.5 months, we observed an overall response rate (ORR) of 59.3% and a median progression-free survival (PFS) of 8.7 months. In subgroup analyses, we found significantly lower ORR and median PFS in patients with extramedullary disease (37%/2.1 months), and/or an ISS of 3 (37%/1.3 months), and ide-cel pretreated patients (33%/1.8 months). Nonetheless, the duration of response in ide-cel pretreated patients was comparable to that of anti-BCMA naive patients. Infections and grade ≥3 cytopenias were the most frequent adverse events. In summary, we found that teclistamab exhibited a comparable efficacy and safety profile in the real-world setting as in the pivotal trial.
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http://dx.doi.org/10.1038/s41375-024-02154-5 | DOI Listing |
Genet Med
December 2024
Movement Disorders Program, Department of Neurology and F.M. Kirby Neurobiology Center, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA. Electronic address:
Objectives: Biallelic HPDL variants have been identified as the cause of a progressive childhood-onset movement disorder, with a broad clinical spectrum from severe neurodevelopmental disorder to juvenile-onset pure hereditary spastic paraplegia type 83. This study aims at delineating the geno- and phenotypic spectra of patients with HPDL-related disease, quantitatively modelling the natural history, and uncovering genotype-phenotype associations.
Methods: A cross-sectional analysis of 90 published and one novel case was performed, employing a Human Phenotype Ontology-based approach.
Orthod Craniofac Res
December 2024
Department of Orthodontics, Faculty of Dentistry, Mahidol University, Bangkok, Thailand.
Objectives: To investigate maxillary canine movement accuracy and anchorage during space closure in first premolar extraction cases (maximum anchorage) using In-House Clear Aligners (IHCAs).
Materials And Methods: A randomised controlled trial with a split-mouth design recruited 16 adults in university setting. Each patient was randomly assigned by side for canine retraction using 12 IHCAs to both the experimental palatal power arm (Pa) and non-Pa control (C).
Nurs Open
January 2025
Department of Nursing, Zhongshan Hospital Xiamen University, Xiamen, China.
Aim: We explored demoralisation syndrome among post-operative patients with breast cancer and its relationship with patients' body image and marital intimacy.
Design: A cross-sectional study.
Methods: In this cross-sectional study, 237 patients with breast cancer who were hospitalised in the breast surgery department of Grade A tertiary hospital in Xiamen, China from June 2022 to December 2023 and met the standards of adaxation were selected by the convenience sampling method.
Orthod Craniofac Res
December 2024
Department of Orthodontics & Dentofacial Orthopaedics, Manav Rachna Dental College, Faridabad, India.
Objective: The study compares and evaluates planned virtual outcomes with actual post-treatment outcomes to assess the accuracy and predictability of clinical results during presurgical infant orthopaedics (PSIO) with AlignerNAM in infants with unilateral cleft lip and palate.
Setting: Institutional study.
Participants: 14 UCLP patients.
Ann Med
December 2025
Department of Hematology, Affiliated Hangzhou First People's Hospital, Westlake University, School of Medicine, Hangzhou, China.
Background: The management of high-risk acute myeloid leukaemia (AML) remains challenging, highlighting the need for innovative conditioning strategies beyond current regimens.
Methods: In the present single-arm study, a FACT regimen comprised of low-dose total body irradiation (TBI) with fludarabine, cytarabine and cyclophosphamide was employed to treat cytogenetically high-risk AML patients exhibiting pre-transplant active disease. This clinical trial is registered in the Chinese Clinical Trial Registry with the registration number ChiCTR2000035111.
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