BackgroundRimegepant is a calcitonin gene-related peptide (CGRP) receptor antagonist approved for both acute and preventive treatment of episodic migraine. Real-world data on its preventive use remain limited, particularly in patients with multiple prior preventive failures. This study evaluated the effectiveness and tolerability of rimegepant in routine clinical practice, focusing on a highly treatment-resistant population.MethodsWe conducted a prospective, multicenter real-world cohort study within the GEMA (GEpants in MigrAine) Project across nine tertiary Headache Units in Spain. Adults initiating rimegepant for migraine prevention were consecutively enrolled and followed for up to 6 months. The primary endpoint was the 3-month change in monthly headache days (MHD). Secondary endpoints included the change in monthly migraine days (MMD), response rates, predictors of response, and tolerability. Baseline characteristics, prior preventive failures, medication overuse, adverse events, and patient-reported outcomes (Headache Impact Test-6 (HIT-6), HADS, and Insomnia Severity Index) were recorded.ResultsIn total, 150 patients completed 3-month follow-up and 64 reached 6 months. The cohort was predominantly female (85.3%), with 70.7% episodic migraine, a median age of 48 years (interquartile range (IQR) = 39-57), and a median of 6 prior preventive failures (IQR = 4-8), reflecting high treatment resistance. At 3 months, median MHD decreased from 12 to 7.5 and MMD from 10 to 6 (p < 0.05), with significant improvement in HIT-6. Overall, 36% and 43% achieved ≥ 50% reduction in MHD and MMD, respectively (≥ 75%: 15% and 20%). Among patients with 6-month data, further reductions were observed (MHD, 6 days; MMD, 5 days), with ≥ 50% response rates increasing to 48% and 58%. Clinical responders showed greater improvements in anxiety and depressive symptoms. Medication overuse, chronic migraine, and prior exposure to anti-CGRP monoclonal antibodies and onabotulinumtoxinA were independent predictors of poorer outcomes, with response declining with increasing prior anti-CGRP exposure, although a relevant proportion still achieved meaningful benefit. Rimegepant was well tolerated, with predominantly mild adverse events (nausea 13%, constipation 8%) and low discontinuation (7% at 3 months), and with nausea being the most frequent cause.ConclusionsRimegepant showed meaningful preventive effectiveness and good tolerability in routine clinical practice, including in highly treatment-resistant patients with prior anti-CGRP monoclonal antibody exposure. The response was influenced by baseline disease burden and prior treatment exposure. These findings suggest that earlier use of rimegepant in the treatment course may be associated with greater clinical benefit.
INTRODUCTION:The standard first-line treatment for metastatic small-cell lung cancer (SCLC) is platinum-based chemotherapy in combination with etoposide. The aim of this study was to evaluate the real-world effectiveness and safety of atezolizumab plus chemotherapy in SCLC and to explore factors associated with survival. METHODS:A retrospective, observational, multicenter study was conducted in patients diagnosed with SCLC who received first-line treatment with atezolizumab in combination with platinum-etoposide chemotherapy between November 2021 and March 2025. Treatment effectiveness was evaluated by assessing the objective response rate (ORR), treatment duration, progression-free survival (PFS) and overall survival (OS). Univariate and multivariate Cox models were used to explore associations between clinical variables and survival outcomes. RESULTS:A total of 76 patients with SCLC were included. Median age was 65 years, and 17.1% had an Eastern Cooperative Oncology Group performance status (ECOG PS) ≥2. The median duration of the treatment was 4.43 months (Interquartile range: 0.4-6.2). The ORR was 81.67% (95% CI: 70.08-89.44%). The median PFS was 7.2 months (95% CI: 6.5-8.6), and the median OS was 9.4 months (95% CI: 7.0-13.06). Patients with ECOG ≥2, hepatic metastases, and age ≥ 65 years were associated with a poor survival prognosis. Sex and the presence of brain metastases were not significantly associated with survival. Grade ≥ 3 treatment-related adverse events were observed in 44.7% patients, with no treatment-related deaths. CONCLUSIONS:In this real-world cohort, atezolizumab plus platinum-etoposide achieved high response rates and acceptable survival with a manageable safety profile in extensive-stage SCLC. Baseline ECOG performance status, hepatic metastases, and older age were associated with worse survival, underscoring the prognostic value of clinical factors. These observational data complement, but do not validate, the results of IMpower133, as no randomized control group or formal trial emulation methods were used.
BACKGROUND:Beta-thalassemia is a genetically heterogeneous hemoglobinopathy with marked clinical variability. In Spain, comprehensive nationwide data on transfusion-dependent beta-thalassemia (TDT) remain limited, particularly regarding molecular characterization. METHODS:This observational study analyzed data from the National Thalassemia Registry of the Spanish Society of Hematology and Hemotherapy (SEHH). Between November 2022 and February 2025, 147 patients with beta-thalassemia from 42 hospitals were registered; this report focuses on 78 patients with TDT. Clinical, transfusion, and biochemical data were collected, and molecular analysis of the HBB and HBA genes was performed using Sanger sequencing, next-generation sequencing, and complementary techniques. Genotypes were classified according to the degree of beta-globin synthesis reduction. RESULTS:The mean age of TDT patients was 34.3 years, and 73.1% were of Spanish origin. Patients received a mean of 31.4 packed red blood cell units per year. Splenectomy had been performed in 35.9% of cases. Most patients showed adequate iron overload control, with median serum ferritin levels below 1000 ng/mL. Twenty-four different HBB mutations were identified; the most frequent were CD39 (C > T), IVS-1-nt1 (G > A), IVS-1-nt110 (G > A), IVS-1-nt6 (T > C), and IVS-1-nt1 (G > T), accounting for 75% of alleles. Genotype distribution was 55.2% β0/β0, 30.3% β0/β+, and 14.4% β+/β+. Patients with β+/β+ genotypes had significantly lower ferritin levels. CONCLUSIONS:This nationwide registry highlights the genetic and clinical heterogeneity of TDT in Spain and underscores the value of molecular characterization for patient management, genetic counseling, and future therapeutic strategies.
Introducción La prevalencia del cáncer ha aumentado asociada a una mayor complejidad de los pacientes. En este contexto, la implantación del internista en la asistencia al paciente con tumor sólido ha crecido en los últimos años. Se pretende describir la actividad del internista en la asistencia al paciente oncológico en los hospitales españoles. Material y métodos Estudio transversal y descriptivo a partir de una cohorte formada por todos los hospitales públicos y los principales privados. La información se recogió a través de encuesta respondida por médicos internistas y oncólogos en enero y febrero de 2025. Resultados Se obtuvo respuesta de 188 de los 481 centros objetivo (39,1%), representando el 51,2% de los centros públicos y el 16,7% de los privados. En 181 centros se atendían pacientes oncológicos hospitalizados, siendo responsabilidad de oncología médica en el 56,4% de los centros y de medicina interna en el 43,6%. Entre aquellos donde ingresaban a cargo de oncología médica, se disponía de internista en planta de hospitalización (modelo de asistencia compartida) en el 37,3% de los casos. En global, en el 64,5% de los centros, los pacientes oncológicos ingresaban en plantas con presencia de internistas, ya fuera mediante atención directa por medicina interna o bien con el modelo de asistencia compartida. Conclusión Los internistas desempeñan un papel esencial en la atención del paciente oncológico en España, especialmente en las plantas de hospitalización. La elevada implantación del internista justificaría su reconocimiento como actividad estructural en la asistencia al paciente oncológico, así como su participación activa en equipos de trabajo multidisciplinares.
