Treatment Outcomes In Patients Research Articles

Petrous bone cholesteatoma (PBC): report of 94 cases of PBC with focus on intraoperative endoscopic assistance, and systematic review of the literature.

The aim of this study is to assess the clinical characteristics, classification, surgical approaches, interventional strategies, and treatment outcomes of patients with petrous bone cholesteatoma (PBC). Observational retrospective study of patients with PBC managed by the senior author of this paper between 1995 and 2024 in a tertiary referral center. A literature review was made, identifying 16 articles. Clinical presentations, Sanna's classification, pre- and post-surgical facial function and audiometry, surgical approach and intraoperative endoscopic assistance, postoperative complications, and recurrence rates were reported. We report on a large series of 94 cases managed by the senior author. The most common type was supralabyrinthine PBCs (40.43%; 38 cases), followed by massive (18.09%; 17). Hearing loss was present in almost all the patients (98.91%). The most frequent approaches were the modified translabyrinthine approach (24.51%; 25), the subtotal petrosectomy (22.55%; 23) and the transotic approach (18.63%; 19). In 11 patients the approach was assisted by the use of an endoscope. Recurrence of PBC was identified in 8 cases (8.51%) that were surgically resolved. Additionally, we report a review of the literature (16 articles). We focus on the introduction of endoscopic assisted techniques that may help in reducing the rate of residual and avoid rerouting of the facial nerve. At the same time, we encourage techniques that include elimination of the middle ear through a subtotal petrosectomy, as they are essential to protect vital structures that have been exposed by the disease or by the surgical technique.

Nebulised high-dose corticosteroids as add-on therapy for adults with asthma exacerbation: a randomised controlled trial

BackgroundEvidence regarding high-dose inhaled corticosteroids (HDICS) in asthma exacerbations in adults is insufficient. This study compares the treatment outcomes of HDICS as add-on therapy to the outcomes of standard treatment...

Congenital hyperinsulinism in the Ukraine: a 10-year national study

IntroductionCongenital Hyperinsulinism (CHI) has not been previously studied in Ukraine. We therefore aimed to elucidate the genetics, clinical phenotype, histological subtype, treatment and long-term outcomes of Ukrainian patients with CHI.MethodsForty-one patients with CHI were recruited to the Ukrainian national registry between the years 2014-2023. Genetic testing (n=40), 18F-fluorodihydroxyphenylalanin and 68Ga-DOTANOC PET/CT imaging followed by surgical treatment and subsequent histological analysis (n=19) was performed through international collaboration.ResultsPathogenic variants were identified in 19/22 (86.3%) individuals with persistent CHI (p-CHI) and 8/18 (44.4%) with early remission CHI (er-CHI). Pathogenic variants in the K-ATP channel genes were the only identified genetic cause of p-CHI (ABCC8 (n=17) and KCNJ11 (n=2)) with greater genetic heterogeneity observed in those with er-CHI (ABCC8 (n=3), KMT2D (Kabuki Syndrome, n=1), Beckwith-Wiedemann syndrome (n=2) and INSR (Donohue syndrome (n=2)). Histological analysis performed on 19 children with persistent CHI confirmed focal disease in 14 (73.7%), diffuse disease in two (10.5%) and atypical histology in three (15.8%). After surgery, complete recovery was observed in all 14 with focal disease, while relapse occurred in three patients with diffuse or atypical histology.ConclusionA genetic diagnosis was achieved for 67.5% (27/40) of the cohort with a higher pick-up rate observed in those with p-CHI. The genetics and imaging studies enabled subtype-targeted treatment with surgical cure achieved in all individuals with focal disease.

Phase I-IIa clinical trial to evaluate the safety, feasibility and efficacy of the use of a palate mucosa generated by tissue engineering for the treatment of children with cleft palate: the BIOCLEFT study protocol

IntroductionThe current gold standard treatment for patients with orofacial clefts is surgical repair of the palatal defect (uranostaphylorrhaphy), which is associated with growth defects and hypoplasia of the maxillofacial structures....

Evaluation of the clinical efficacy of modified LSC transplantation plus BCL implantation in the treatment of pterygium

ObjectiveTo explore the clinical efficacy of modified limbal stem cell transplantation(Modified LSC transplantation) and bandage contact lens(BCL) implantation in pterygium surgery.MethodsA total of 479 patients with primary pterygium who were admitted to our hospital from March 2019 to March 2023 were randomly divided into three groups: the normal group (Group A: 89 patients), the control group (Group B: 195 patients), and the modified group (Group C: 195 patients). Each group received different intervention measures. Group A did not undergo surgical treatment and were required to follow up as outpatients. Group B received LSC transplantation combined with interrupted suturing plus BCL, whereas Group C received modified LSC transplantation combined with BCL. The degree of corneal irritation symptoms, wound healing and graft status under slit lamp, incidence and recurrence rate of complications, tear film rupture time, tear secretion test, intraocular pressure, ocular surface inflammation response(IL-1β, PGE2, TNF-α, VEGF), and visual quality were compared and analyzed at various time points after surgery.ResultsCompared with those in the Group B, patients in the Group C experienced faster normalization of corneal epithelium recovery, fewer corneal irritation symptoms, and better wound healing. The break-up time (BUT) of the tear film at 1 week to 1 year postoperatively was significantly greater in the Group C than Group B, with values approaching those of Group A by 3 months (P < 0.05). The Schirmer test results revealed a similar trend to that of the BUT. Further analysis of intraocular pressure (IOP) at different time points revealed no significant differences among the three groups at postoperative Day 1. However, due to the use of corticosteroid eye drops postoperatively, IOP was greater in both the Group B(17.24 ± 2.12 mmHg) and Group C (17.02 ± 2.37 mmHg) than Group A (13.92 ± 1.57 mmHg) at 1 week. By 1 month, Group C had a lower IOP (15.77 ± 1.63 mmHg) than Group B(17.78 ± 2.41 mmHg). There were no significant differences in IOP among the three groups from 3 months to 1 year (P > 0.05). The ELISA results indicated that the expression levels of the ocular surface inflammatory factors IL-1β, TNF-α, PEG2, and VEGF in the Group C were lower than those in Group B from 1 week to 1 year post surgery. Under both natural light and low-light conditions (spatial frequency/6 cd), Group C had better best-corrected visual acuity and contrast sensitivity than Group B at 1 week to 1 year postoperatively. Additionally, Group C had lower corneal higher-order aberrations (including astigmatism, spherical aberrations, and total higher-order aberrations) and superior vision-related quality of life scores at 1 year postoperatively than Group B, with statistically significant differences (P < 0.05).ConclusionModified LSC transplantation combined with BCL implantation provided superior treatment outcomes for patients with pterygium, which was worthy of further clinical promotion.

Characteristics and treatment outcomes of patients with multi-drug-resistant tuberculosis at Abbassia Chest Hospital

BackgroundMulti-drug-resistant tuberculosis (MDR-TB) remains a significant health concern. This study analyzes patients admitted to Abbassia Chest Hospital, focusing on their characteristics, drug resistance patterns, treatment outcomes, and risk factors for poor prognosis.MethodsA retrospective cohort study was conducted on 121 patients admitted to the MDR unit at Abbassia Chest Hospital. TB diagnosis was confirmed using sputum smear microscopy, microbiological culture and sensitivity testing, and molecular methods (GeneXpert). Patient data were extracted from hospital records for statistical analysis.ResultsThe mean age of MDR-TB patients was 40 years, with 75.21% being male. Key risk factors included prior TB interrupted treatment, smoking, and contact with MDR-TB patients. Resistance rates exceeded 90% for cycloserine, ethionamide, pyrazinamide, and levofloxacin, while 71.9% were resistant to amoxicillin. Sputum conversion occurred in 95.04% of patients, with a mean conversion time of 2.21 ± 1.44 months. Successful treatment was achieved in 84.3% of cases, while 45.45% experienced side effects, the most common being hyperuricemia (28.1%), gastritis, and ototoxicity (11.57% each). Bad prognosis was associated with comorbidities like ischemic heart disease (OR 15.34, p = 0.02) and delayed sputum conversion (OR 1.63/month, p = 0.03). Side effects were not linked to bad outcomes.ConclusionWhile most MDR-TB patients achieved successful treatment outcomes, certain factors, such as comorbidities (particularly ischemic heart disease) and delayed sputum conversion, significantly contributed to bad prognosis.

Clinical Outcome of Intravenous Alteplase Treatment in Patients With Acute Mild Cerebral Infarction

ABSTRACTBackgroundThe efficacy of intravenous alteplase therapy for acute cerebral infarction is well established, regardless of the ischemic stroke subtype. However, its effectiveness in patients with mild cerebral infarction remains controversial, raising concerns about the significance of the treatment.AimTo retrospectively evaluate the efficacy of intravenous alteplase in patients with mild cerebral infarction and other factors affecting the outcome.MethodsA total of 527 patients received intravenous alteplase (0.6 mg/kg) at our hospital between October 2012 and March 2023. Of these, 118 patients with mild cerebral infarction (National Institutes of Health Stroke Scale [NIHSS] ≤ 5) who were treated with intravenous alteplase without mechanical thrombectomy were retrospectively evaluated for treatment efficacy and the factors affecting the efficacy.ResultsThe modified Rankin scale (mRS) outcome at 90 days was good (mRS: 0–1) in 85 (72%) patients and poor (mRS: 2–5) in 33 (28%) patients. Branch atheromatous disease (BAD) and proximal anterior circulation large vessel occlusion (proximal LVO: internal carotid artery and M1 segment of middle cerebral artery) were observed more frequently in the poor outcome group than in the good outcome group. Multivariate analysis, adjusted for age and sex, identified BAD and proximal LVO as factors associated with poor outcomes after intravenous alteplase treatment for mild cerebral infarction. Symptomatic intracranial hemorrhage was observed in one patient with BAD.ConclusionMost patients with mild cerebral infarction who were treated with intravenous alteplase alone had good outcomes. BAD and proximal LVO were other factors influencing poor outcomes.

The effect of transition timing of regorafenib on treatment outcomes in unresectable hepatocellular carcinoma: a real-world study.

Regorafenib is the recommended second-line therapy for unresectable hepatocellular carcinoma (uHCC). Our study aimed to assess the effect of transition timing to second-line therapy with regorafenib on treatment outcomes in uHCC patients. In this retrospective study, patients were categorized into prompt transition group (PT group) or delayed transition group (DT group) based on the transition timing to second-line therapy with regorafenib following the first or subsequent occurrence of progressive disease during first-line treatment. The primary endpoint was progression-free survival (PFS), and the secondary endpoints were objective response rate (ORR), disease control rate (DCR), overall survival (OS), and safety. There were 85 and 122 patients in the PT and DT groups, respectively. The PT group demonstrated significantly better median PFS [4.5months (95% CI 3.8-6.0) vs. 3.4months (95% CI 2.8-4.1); HR 0.641; 95% CI 0.478-0.859; p = 0.003], ORR (24.7% vs. 9.8%, p = 0.007), DCR (69.4% vs. 54.9%, p = 0.049), and median OS [17.5months (95% CI 13.4-not reached) vs. 10.4months (95% CI 7.9-15.6); HR 0.613; 92% CI 0.432-0.871, p = 0.006] compared to the DT group. Moreover, the overall safety profiles were comparable between groups. The prompt transition to second-line therapy with regorafenib following first-line treatment progression suggests better treatment outcomes and potentially longer survival than in patients who delay the transition to second-line therapy.

Synergistic potential of lopinavir and azole combinational therapy against clinically important Aspergillus species.

Aspergillus fumigatus is a widely distributed pathogen responsible for severe infections, particularly in immunocompromised individuals. Triazoles are the primary treatments options for Aspergillus infections; however, the emergence of acquired resistance to this antifungal class is becoming a growing concern. In this study, we investigated the potential of the antiviral drug, lopinavir (LPV) to restore the susceptibility of A. fumigatus strains to a set of azoles, while also reducing the required azole dosage for treatment of susceptible isolates. The combination of LPV with either itraconazole (ITC) or posaconazole (POS) demonstrated potent synergistic interactions against 16 out of 23 (~70%) and 21 out of 23 (~91%) A. fumigatus isolates, respectively. Moreover, the combination showed synergistic activity against other clinically important Aspergillus species, including A. niger, A. flavus, and A. brasiliensis. The fractional inhibitory concentration index (FICI) for the combinations ranged from 0.18 to 0.313 for ITC and 0.091 to 0.313 for POS, indicating strong synergistic effects. Further investigation revealed that efflux pump inhibition contributed to the synergy observed between azole and LPV. Morphological examination of the fungal cells subjected to this combinational therapy at sub-inhibitory doses showed the presence of carbohydrate granules/patches. The identification of LPV as a promising adjunct therapy holds promise for addressing the emerging challenge of azole resistance in Aspergillus species and improving treatment outcomes for patients.

Cutibacterium acnes periprosthetic joint infections.

Cutibacterium acnes (C. acnes; previously known as Propionibacterium acnes or P. acnes) periprosthetic hip and knee infections are under-reported. While culture contamination with C. acnes occurs, true infections are important to recognize and treat. We sought to describe the demographics and treatment outcomes of patients with C. acnes periprosthetic joint infections (PJIs) of the hip and knee. Patients with C. acnes PJI between January 2005 and December 2018 were retrospectively reviewed utilizing the institutional total joint registry. Patients with monomicrobial PJI and two or more positive cultures were considered to have true C. acnes PJI. Patients with polymicrobial infection or with only one positive culture were excluded. This resulted in 35 PJIs (21 hips and 14 knees); the patients' mean age was 63 years (35 to 84) and 15 (43%) were female. Mean follow-up was five years (1 to 14). The median time to positive culture was five days (IQR 5 to 6) and median synovial fluid cell count was 22,583 cells (IQR 15,200 to 53,231). The median ESR was 25 mm/hr (IQR 7 to 37), and CRP was 15 mg/l (IQR 3 to 29). Of the 35 PJIs, 18 (51%) were treated with chronic antibiotic suppression without surgical intervention, and the remainder were treated with two-stage exchange arthroplasty. The two-year survival free of any revision was 94%. Four patients failed treatment due to symptomatic infection, with three treated with two-stage exchange and one treated with irrigation and debridement with modular component exchange for a survival rate of 89% and 83% at two and five years, respectively. Laboratory evidence of C. acnes PJI in this cohort was typical compared to more conventional organisms. Cultures grew more quickly than previously thought in patients with C. acnes PJI. Treatment with two-stage exchange or chronic antibiotic suppression alone both had few treatment failures at mid-term follow-up.

Destombes-Rosai-Dorfman disease: A case series of nodular and extra nodular involvement

Introduction and importanceRosai-Dorfman disease (RDD) is a rare benign histiocytic proliferative disorder characterized by painless lymph node enlargement and extranodal involvement. This disease often presents diagnostic challenges due to its variable clinical manifestations and the need for histologic confirmation. Case series presentationThis case series examines five patients diagnosed with Rosai-Dorfman disease who presented with a variety of clinical manifestations. Patients ranged in age from 21 to 72 years and presented with a variety of symptoms including cervical lymphadenopathy, mediastinal adenopathies, thyroid nodules, and nasal obstruction. Diagnoses were confirmed by histologic examination and immunostaining, primarily with anti-PS100 antibody. Treatments included surgery, corticosteroids, and laser resection, with most cases showing favorable outcomes and no recurrence at follow-ups ranging from one to five years. Clinical discussionThe diverse presentations of RDD in this case series underscore the importance of considering this entity in the differential diagnosis of lymphadenopathy and other mass lesions. Histologic examination and immunostaining are critical for accurate diagnosis. ConclusionThis case series aims to highlight the diverse presentations, diagnostic approaches, and treatment outcomes of patients with Rosai-Dorfman disease and to contribute to a better understanding of this rare condition.

Efficacy and safety profile of 2nd line Anti TB drugs in DRTB patients in a tertiary care hospital of South Punjab.

The objective of the study was to assess the efficacy and safety profile of 2nd line Anti TB drugs in DR TB patients in a tertiary care hospital of South Punjab. This retrospective cohort study included patients who received therapy for multidrug-resistant tuberculosis (MDR-TB) between March 2018 and June 2021 at the Pulmonology Department of Sheikh Zayed Hospital, Rahim Yar Khan. Sociodemographic data, TB treatment history, treatment schemes, safety profiles, weight measures, and sputum smear results were obtained from medical records. Outcome variables of interest included body weight, sputum smear grading for treatment efficacy, and ADRs for safety assessment. The study showed a progressive decline in positive sputum smear results over the 24-month treatment for MDR-TB, with an increase in negative smear results from 33 (9.50%) at baseline to 97 (28.0%) by Month 24. Furthermore, the mean body weight significantly increased every three months throughout the trial. Beginning at 43.83 kg at baseline, the mean weight increased to 50.02 kg by the end of 24 months. ADRs were mostly mild to moderate, including depression (18.4%), arthralgia (17.1%), anxiety (16.5%), and Qtc prolongation (11.0%). The treatment adhered to international guidelines, using a tailored combination of second-line drugs and adjusting regimens based on drug resistance patterns from sputum culture and DST. The study findings suggest the efficacy of second-line anti-TB drug regimens in achieving sputum smear conversion and positive treatment outcomes in patients with MDR-TB. However, the occurrence of ADRs highlights the need for careful monitoring and management during treatment.

Gastrointestinal Neuroendocrine Tumors-Case Series from a Tertiary Care Center with Review of Literature.

Neuroendocrine tumors account for 0.5% of all malignancies, and their incidence is around 2/100,000 with a female predilection within the age range of 50years at the time of presentation. NETs in India have a different clinicopathological profile compared to those in Western countries. Very few limited studies are available in Indian literature with regard to the epidemiology, presentations, histopathological characteristics, and treatment outcomes in patients with gastrointestinal neuroendocrine tumors. This warrants the need for documentation of these tumors, and we hereby present a case series of four patients diagnosed with NETs who presented within a period of 9months at our rural tertiary care center.

Stability and outcome of surgical-orthodontic treatment in patients of Class III deformity with and without a history of orthodontic treatment

Stability and outcome of surgical-orthodontic treatment in patients of Class III deformity with and without a history of orthodontic treatment

Clinical, antibiotic resistance features, and treatment outcomes of Vietnamese patients with community-acquired sepsis caused by Klebsiella pneumoniae

Clinical, antibiotic resistance features, and treatment outcomes of Vietnamese patients with community-acquired sepsis caused by Klebsiella pneumoniae

Contribution of Scalp Regions to Machine Learning-Based Classification of Dementia Utilizing Resting-State qEEG Signals.

This study aims to investigate using eyes-open (EO) and eyes-closed (EC) resting-state EEG data to diagnose cognitive impairment using machine learning methods, enhancing timely intervention and cost-effectiveness in dementia research. A total of 890 participants aged 40-90 were included in the study, comprising 269healthy controls (HC), 356 individuals with mild cognitive impairment (MCI), and 265 with Alzheimer's disease (AD) from a cohort study. Resting-state EEG (rEEG) signals were recorded and transformed into relative power spectral density (PSD) data for analysis. The processed PSD data, representing 19 scalp regions, were then input into a Random Forest (RF) machine learning classifier to identify distinctive EEG patterns across the groups. Statistical comparisons between the groups were conducted using one-way ANOVA, applied to the relative PSD features extracted from the EEG data, to assess significant differences in EEG activity across the diagnostic categories. The study found that rEEG-based categorization effectively differentiates between cognitively impaired individuals and healthy individuals. The EO rEEG achieved the highest performance metrics across various models. For HC vs MCI (combined hemisphere), the accuracy, sensitivity, specificity, and AUC were 92%, 99%, 83%, and 96%, respectively. For HC vs AD (parietal, temporal, occipital), these metrics were 95%, 96%, 94%, and 99%. The HC vs CASE (MCI + AD) (combined hemisphere) results were 90%, 99%, 73%, and 92%. The metrics for HC vs MCI vs AD (frontal, parietal, temporal) were 89%, 88%, 94%, and 96%. The study demonstrates that EO rEEG can effectively distinguish between cognitive impairment and healthy states, leading to early diagnosis, cost-effective treatment, and better clinical outcomes for dementia patients. EO and EC rEEG models trained with relative PSD, particularly from parietal, temporal, occipital, and central scalp regions, can significantly assist clinicians in practice.

An evolving machine-learning-based algorithm to early predict response to anti-CGRP monoclonal antibodies in patients with migraine.

The present study aimed to determine whether machine-learning (ML)-based models can predict 3-, 6, and 12-month responses to the monoclonal antibodies (mAbs) against the calcitonin gene-related peptide (CGRP) or its receptor (anti-CGRPmAbs) in patients with migraine using early predictors (up to one month) and to create an evolving prediction tool. In this prospective cohort study, data from patients with migraine who had received anti-CGRP mAbs for 12 months were collected. Demographic and monthly clinical variables were collected, including monthly headache days (MHDs), days with acute medication use, number of analgesics and Headache Impact Test-6. Response rates were categorized as <25%, 26-50%, 51-75% and >75% reduction in MHDs. ML models were trained using random forest algorithm and optimized to maximize the F1 score. ML model performance was also evaluated using standard evaluation metrics, including accuracy, precision and area under the receiver operating characteristic curve (AUC-ROC). Sequential backward feature selection was employed to identify the most relevant predictors for each model. Each model was given 11 baseline data inputs and month-based predictors for months 1, 3 and 6. Each model was then validated against an external test cohort of patients who had received anti-CGRP mAbs for 12 months. Three hundred thirty-six patients treated with anti-CGRP mAbs were included. The external cohort included 93 patients treated with anti-CGRP mAbs. We developed six models to predict 3- 6- and 12-month responses using early predictors. ML-based models yielded predictions with an accuracy score in the range 0.40-0.73 and an AUC-ROC score in the range 0.56-0.76 during internal testing and yielding predictions with an accuracy in the range 0.39-0.64 and an AUC-ROC score in the range 0.52-0.78 when tested against an external test cohort. Shapley Additive explanations summary plots were generated to interpret the contribution of each feature for each model. Based on these findings, a response prediction tool was developed. Each model was run through a backward feature selection to find the most relevant features for the models. The MHDs reduction of the previous data point tends to be the most relevant, while the migraine with aura indicator tends to be the least effective predictor. The response prediction tool utilizing evolving ML-based models holds promise in the early prediction of treatment outcomes for patients with migraine undergoing anti-CGRP mAbs treatment.

Sex differences in patients with atrial fibrillation and acute coronary syndrome or undergoing PCI: a real-world study

Background In patients with acute coronary syndrome (ACS) or undergoing percutaneous coronary intervention (PCI), female patients have a worse short-term prognosis than male patients has been consistently concluded in many studies. However, the impact of sex differences on long-term prognosis remains uncertain. Additionally, women are underrepresented in clinical trials exploring optimal antithrombotic strategies in patients with atrial fibrillation (AF) and ACS or PCI. To bridge this gap, this study aimed to investigate sex differences in clinical characteristics, treatment, and long-term clinical outcomes in patients with AF and ACS or PCI. Patients and methods This study included a total of 1237 patients with AF and ACS and 539 patients with AF and stable coronary artery disease (SCAD) who underwent PCI at the Fuwai Hospital of the Chinese Academy of Medical Sciences from January 2017 to December 2019. Patients were followed up until the end of 2021 to observe the occurrence of major adverse cardiovascular events (MACE). The relationship between sex and MACE was evaluated using Cox regression models and Kaplan–Meier’s survival curves. Results In patients with AF and ACS, multivariable COX regression analysis revealed that female patients were independently associated with a higher risk of MACE (HR = 1.45, 95% CI 1.11–1.89, p = .006). However, in AF patients with SCAD who underwent PCI, the analysis showed that female patients were not independently associated with MACE risk (HR = 1.12, 95% CI 0.62–2.03, p = .717). Conclusions In patients with AF and ACS, females have a significantly higher risk of long-term MACE compared to males. However, among patients with AF and SCAD who underwent PCI, there was no significant difference in long-term MACE risk between males and females.

Overlap syndrome of Erdheim-Chester disease and Langerhans cell histiocytosis: A case report

Langerhans cell histiocytosis (LCH) and Erdheim–Chester disease (ECD) are exceptionally rare disorders characterized by varied clinical presentations, posing several challenges for clinicians. The concomitant occurrence of LCH and ECD is exceedingly rare and has no known etiology. In this report, we present a rare case of mixed histiocytosis (both ECD and LCH) with multisystem involvement. The patient, a 49-year-old female, initially presented with a rash 2 years ago and progressively developed exophthalmos, fatigue, and shortness of breath. She lacked the mutation in codon 600 of exon 15 of B-Raf proto-oncogene (BRAF-V600E) and subsequently underwent treatment with corticosteroids, interferon-alpha, and chemotherapy, all of which proved ineffective. This work highlights the urgent need to improve treatment outcomes for such patients. Therefore, we discuss the latest advancements in understanding treatment strategies for mixed histiocytic syndromes.

Immunotherapy Following Anaplastic Lymphoma Kinase Inhibitor Therapy for Patients with Anaplastic Lymphoma Kinase‑Positive Non‑small Cell Lung Cancer in Japan.

Although anaplastic lymphoma kinase inhibitors (ALKis) are the effective initial treatment for patients with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC), most patients experience resistance to ALKis, leading to the need for alternative therapies. Immune checkpoint inhibitors (ICIs) are a standard NSCLC treatment. On the other hand, their efficacy remains unclear for ALK-positive NSCLC. We aim to describe the treatment patterns and treatment outcomes for patients with ALK-positive NSCLC receiving later-line ICI treatment. This retrospective cohort study used claims data from Japanese acute care hospitals and included patients with lung cancer (International Classification of Diseases, 10th version (ICD-10), code: C34) diagnosed between 1 December 2015 and 31 January 2023. We extracted patients who received ALKis as first-line therapy and subsequent lines of treatment. Patient characteristics and treatment patterns and durations were descriptively summarized. Time to treatment discontinuation (TTD) for ICIs was examined using Kaplan-Meier estimates. Of 478 patients who received ALKi as first-line treatment, 30 received ICIs, 249 ALKis, and 154 non-ICI/ALKi therapy as second-line treatment. Most patient characteristics showed no differences among the groups. ICIs were more likely to be administered to patients who underwent shorter durations of ALKi treatment. The median TTD for ICIs was 66 days, with a 1 year TTD rate of 13%. Given the rarity of ALK-positive NSCLC, this study contributes to add evidence through an expanded database and increased sample size, supporting previous suggestions that ICIs have limited effectiveness in patients positive for ALK.