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1.
Sci Rep ; 14(1): 7271, 2024 03 27.
Artigo em Inglês | MEDLINE | ID: mdl-38538905

RESUMO

Myasthenia gravis (MG) is a rare, autoimmune, antibody-mediated, neuromuscular disease. This study analyzed digital conversations about MG to explore unprovoked perspectives. Advanced search, data extraction, and artificial intelligence-powered algorithms were used to harvest, mine, and structure public domain digital conversations about MG from US Internet Protocol addresses (August 2021 to August 2022). Thematic analyses examined topics, mindsets, and sentiments/key drivers via natural language processing and text analytics. Findings were described by sex/gender and treatment experience with steroids or intravenous immunoglobulin (IVIg). The 13,234 conversations were extracted from message boards (51%), social media networks (22%), topical sites (21%), and blogs (6%). Sex/gender was confirmed as female in 5703 and male in 2781 conversations, and treatment experience was with steroids in 3255 and IVIg in 2106 conversations. Topics focused on diagnosis (29%), living with MG (28%), symptoms (24%), and treatment (19%). Within 3176 conversations about symptoms, eye problems (21%), facial muscle problems (18%), and fatigue (18%) were most commonly described. Negative sentiments about MG were expressed in 59% of conversations, with only 2% considered positive. Negative conversations were dominated by themes of impact on life (29%), misdiagnosis problems (27%), treatment issues (24%), and symptom severity (20%). Impact on life was a key driver of negativity in conversations by both men (27%) and women (34%), and treatment issues was a dominant theme in conversations by steroid-treated (29%) and IVIg-treated (31%) patients. Of 1382 conversations discussing treatment barriers, 36% focused on side effects, 33% on lack of efficacy, 21% on misdiagnosis, and 10% on cost/insurance. Side effects formed the main barrier in conversations by both steroid-treated and IVIg-treated patients. Capturing the patient voice via digital conversations reveals a high degree of concern related to burden of disease, misdiagnosis, and common MG treatments among those with MG, pointing to a need for treatment options that can improve quality of life.


Assuntos
Imunoglobulinas Intravenosas , Miastenia Gravis , Humanos , Masculino , Feminino , Imunoglobulinas Intravenosas/uso terapêutico , Inteligência Artificial , Análise de Sentimentos , Qualidade de Vida , Miastenia Gravis/diagnóstico , Miastenia Gravis/tratamento farmacológico , Efeitos Psicossociais da Doença , Esteroides
2.
Curr Med Res Opin ; 37(1): 59-70, 2021 01.
Artigo em Inglês | MEDLINE | ID: mdl-33148054

RESUMO

OBJECTIVE: The results from basket trials utilized to gain regulatory approval of tumor-agnostic therapies can be difficult to interpret without the context of a comparator arm. We describe the role and efficacy of histology-based treatments to provide a historical comparison with larotrectinib. METHODS: A systematic literature review (SLR) was conducted on the clinical outcomes of current histology-based standard of care treatments used in non-small cell lung cancer, colorectal cancer, thyroid cancer, gliomas, soft tissue sarcoma, salivary gland cancer, and infantile fibrosarcoma (7 of the 21 tumor histologies in the larotrectinib trials). The review focused on advanced stage/metastatic disease to make a historical comparison with larotrectinib. RESULTS: Larotrectinib provides positive outcomes in both adult and pediatric patients with advanced or metastatic solid tumors known to harbor NTRK gene fusions across a wide range of tumor types. Although the numbers of patients per tumor type are limited, the results of this historical comparison demonstrated that larotrectinib is an efficacious treatment option when naïvely indirectly compared with historical treatments across all 7 reviewed tumor types, especially in comparison to later lines of therapy. CONCLUSIONS: Utilizing larotrectinib as a case example across these types of historical comparisons shows that larotrectinib provides positive efficacy outcomes in TRK fusion cancer across tumor histologies known to harbor NTRK gene fusions that may be preferable to historical treatments.


Assuntos
Neoplasias , Inibidores de Proteínas Quinases/uso terapêutico , Pirazóis/uso terapêutico , Pirimidinas/uso terapêutico , Humanos , Neoplasias/tratamento farmacológico , Neoplasias/patologia , Resultado do Tratamento
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