Patient Treatment Characteristics Research Articles

Treatment characteristics of patients with hereditary transthyretin amyloidosis: a cohort study

BackgroundThere are novel medications approved for the treatment of hereditary transthyretin amyloidosis (ATTRv), classified as transthyretin (TTR) stabilizers or gene silencers. While many patients may be on both classes of medications, there is no data available on the safety and efficacy of combination therapy.ObjectivesTo describe ATTRv patient and TTR-targeted therapy characteristics in a US cohort, and compare outcomes with combination therapy versus monotherapy.MethodsWe performed a retrospective cohort study with electronic health record data of patients with ATTRv seen at a single institution between January 2018 and December 2022. We collected data on symptomatology, gene mutation, disease severity, ATTRv treatment, hospitalizations, and mortality.ResultsOne hundred sixty-two patients with ATTRv were identified. The average age at diagnosis was 65 years. 86 patients (53%) had the V122I variant. 119 patients were symptomatic, of whom 103 were started on ATTRv-specific treatment. 41 patients (40%) had cardiomyopathy only, and 53 (51%) had a mixed phenotype of cardiomyopathy and neuropathy. 38 patients (37%) received therapy with both a gene silencer and protein stabilizer. 9 patients (15%) in the monotherapy group had two or more cardiac hospitalizations after starting treatment, compared to 3 patients (9%) on combination therapy (p=0.26). The adjusted hazard ratio of all-cause mortality for the patients on combination therapy compared to monotherapy was 0.37 (0.08-1.8, p=0.21).ConclusionsWhile the efficacy is unproven, over one-third of patients with ATTRv are on both a stabilizer and a silencer. There were no safety issues for combination therapy. There was a trend towards improved hospitalizations and survival in patients in the combination group but this was not statistically significant. Larger studies with longer follow-up are necessary to determine benefit of combination therapy.

Relationship Between Sepsis Phenotypes and Treatment Characteristics of Patients with Viral and Bacterial Pneumonia

New subgroups of patients with severe community-acquired pneumonia (SCAP) are hardly predicted by the use of clinical covariates; clusterization may significantly improve diagnostic approaches and facilitate the adaptation of specific treatment modalities to patient’s individual characteristics.The aim of the study. To identify linking the sepsis phenotype in patients with SCAP and preferable treatment option to forecasting the outcome and improve treatment results.Materials and methods. Case histories of 664 of intensive care unit (ICU) patients with sepsis (2016–2023) from I. I. Mechnikov Northwestern State Medical University were analyzed. The study included 568 (85.5%) patients with viral SCAP (SCAPv group) and 96 (14.5%) patients with bacterial SCAP (SCAPb group). Sepsis phenotypes were identified using algorithm proposed by Seymour C.W. et al. In SCAP cases associated with COVID-19 infection (n=293, 51.6%) patients received genetically engineered biological therapy (GIBT). The study compared two cohorts of patients: those who received GIBT and did not receive GIBT. Data were statistically processed using the Statistica 10.0 and SPSS software packages.Results. Analysis revealed 4 sepsis phenotypes: α- (N=323, 48.6%); β- (N=128, 19.3%); γ- (N=87, 13.1%); δ - (N=126, 19%). The majority of SCAPv group patients — 295 (51.9%) — had α-phenotype of sepsis, while δ -phenotype prevailed in the SCAPb group — 53 (55.2%). The proportion of patients receiving GIBT and exhibiting α- sepsis phenotype dominated over other sepsis phenotypes: 61.8% of patientspossesed α- phenotype, whereas β-, γ- and δ -phenotypes were determined in 16% , 12.6%, and 9.6% of GIBT patients, respectivelty (P<0.05). The best effect of using monoclonal antibodies to interleukin-6 receptors as a GIBT was obtained in patients with the α-phenotype sepsis and COVID-19-associated SCAP: 87.5% favorable outcomes, P=0.0419. Rate of bacterial sepsis was significantly lower in patients with α- and δ -phenotypes of sepsis receiving GIBT vs those who did not receive this therapy: 12.71% vs 23.2% of patients with α-phenotype, P=0.0131; 25.0% vs 70.41% of patients with δ -phenotype, P=0.0254, respectively.Conclusion. Differences in sepsis phenotype between patients with viral or bacterial SCAP may stratify patients for different therapeutic management and more accurately predict potential complications and unfavorable outcome.

Epidemiological and treatment characteristics of patients with chronic obstructive pulmonary disease on maintenance inhaled therapy between 2011 and 2019 in Hungary

Bevezetés: A krónikus obstruktív tüdőbetegség (COPD) morbiditása és mortalitása a világon és hazánkban is emelkedik. A betegség kezelésének alapja a fenntartó inhalációs terápia és a betegek ezzel szembeni jó adherenciája, de hazánkban a kezelt betegek tekintetében kevés adattal rendelkezünk. Célkitűzés: A COPD miatt 2011 és 2019 között kezelt betegek főbb epidemiológiai és terápiás jellemzőinek meghatározása és ezek összehasonlítása. Módszerek: A Nemzeti Egészségbiztosítási Alapkezelő adatbázisának használatával vizsgáltuk a 2011 és 2019 között fenntartó inhalációs kezelés alatt álló COPD-s betegek adatait (>40 év, az adott évben vagy az azt megelőző bármelyik évben J44-es BNO-kódra felírt fenntartó inhalációs gyógyszert váltott ki minimum 1 alkalommal, melyet kevesebb mint 1 éven belül követett még két további kiváltás). A betegek adatai a bekerülés évétől kezdve minden évben elemzésre kerültek. A betegek korát, nemét, inhalációs kezelési sémáit, a fenntartó inhalációs kezeléssel szembeni adherenciát (gyógyszerkiváltás alapján) és a retard oralis teofillinkészítmények kiváltását hasonlítottuk össze évenként (khi2-próba). Eredmények: Összesen 227 251 beteg adatait elemeztük (2011–2019: 81 308–160 241 fő/év). 2011-ben a 70 év feletti és férfi betegek, míg 2019-ben a 60–69 éves és női betegek voltak többségben. Az egykomponensű, hosszú hatású hörgőtágítót és az inhalációs kortikoszteroidot kiváltó betegek aránya a megfigyelési időszakban csökkent, míg a kettős bronchodilatatorok kiváltása megjelent és fokozatosan emelkedett. A betegek csak valamivel több mint felének esetében (2019: 51,6%) volt jó a fenntartó inhalációs kezeléssel szembeni adherencia (>180 nap/év). Az oralis teofillinkészítményt kiváltók száma nem csökkent (2019-ben a kiváltók aránya: 32%). Megbeszélés: 2011 és 2019 között a fenntartó inhalációs kezelés alatt álló COPD-s betegek száma elmarad a regisztrált betegek számától. A fenntartó inhalációs terápiával szembeni adherencia a betegek jelentős részében nem megfelelő. Az oralis teofillinkészítményeket kiváltók aránya magas. Következtetés: A COPD-s betegek fenntartó inhalációs kezelésével szembeni adherencia javítása szükséges, hogy hazánkban a betegség prognózisa kedvezőbb legyen. Orv Hetil. 2024; 165(9): 338–345.

Demographic, Clinical and Treatment Characteristics of Patients with Hepatocellular Cancer: 10 Years of Experience of a Single Center

Primary cancers of the liver are in the 3rd place in cancer-related deaths. Hepatocellular carcinoma (HCC) accounts for approximately 80% of primary hepatic malignancies. The Barcelona Clinic Liver Cancer (BCLC) classification divides HCC into 5 stages and allocates treatments. The aim of this study is to examine the initial treatments of HCC patients in our center and compare them with the approaches in the guidelines. The data of patients diagnosed with HCC between 2006 and 2016 were recruited retrospectively. Age, gender, etiology, Child-Pugh score, BCLC stage, model for end-stage liver disease score, alpha-fetoprotein level, tumor characteristics, type of first treatment and survival time were evaluated. There were 228 men and 33 women. The mean age was 61±10.5. Of the 130 patients 77 were found on BCLC stage C. The first treatment according to BCLC stage were sorafenib in 22, resection in 16, TACE in 15, Ytrium-90 in 14, systemic chemotherapy other than sorafenib in 14, transplantation in 11, palliative treatment in 8, and ablation in 4 patients. The median survival time was 11.9 (8.1-15.9) months. One-year survival was 32%, three-year survival was 19%, and five-year survival was 16%. The management of HCC patients was performed according to more than just BCLC guidelines in our center. The high number of patients who did not receive treatment may indicate that we need to show sufficient clinical attention to Stage D patients.

Impact of sex on severe asthma: a cross-sectional retrospective analysis of UK primary and specialist care

IntroductionAfter puberty, females are more likely to develop asthma and in a more severe form than males. The associations between asthma and sex are complex with multiple intrinsic and external...

A large single-center cohort of bare lymphocyte syndrome: Immunological and genetic features in Turkey.

Major histocompatibility complex class II (MHC-II) deficiency or bare lymphocyte syndrome (BLS) is a rare, early-onset, autosomal recessive, and life-threatening inborn error of immunity. We aimed to assess the demographic, clinical, laboratory, follow-up, and treatment characteristics of patients with MHC-II deficiency, together with their survival. We retrospectively investigated 21 patients with MHC-II deficiency. Female/male ratio was 1.63. The median age at diagnosis was 16.3 months (5 months-9.7 years). Nineteen patients (90.5%) had parental consanguinity. Pulmonary diseases (pneumonia, chronic lung disease) (81%), diarrhoea (47.6%), and candidiasis (28.6%) were common. Four (19%) had autoimmunity, two developed septic arthritis, and three (14%) developed bronchiectasis in the follow-up. Three patients (14%) had CMV viraemia, one with bilateral CMV retinitis. Eight (38.1%) had lymphocytopenia, and four (19%) had neutropenia. Serum IgM, IgA, and IgG levels were low in 18 (85.7%), 15 (71.4%), and 11 (52.4%) patients, respectively. CD4+ lymphocytopenia, a reversed CD4+/CD8+ ratio, and absent/low HLA-DR expressions were detected in 93.3%, 86.7%, and 100% of the patients, respectively. Haematopoietic stem cell transplantation (HSCT) was performed on nine patients, and four died of septicaemia and ARDS after HSCT. The present median age of patients survived is 14 years (1-31 years). Genetic analysis was performed in 10 patients. RFX5 homozygous gene defect was found in three patients (P1, P4 and P8), and RFXANK (P2 and P14) and RFXAP (P18 and P19) heterozygous gene defects were found in each two patients, respectively. This large cohort showed that BLS patients have severe combined immunodeficiency (SCID)-like clinical findings. Flow cytometric MHC-II expression study is crucial for the diagnosis, differential diagnosis with SCID, early haematopoietic stem cell transplantation (HSCT), and post-HSCT follow-up. Genetic studies are required first for matched family donor evaluation before HSCT and then for genetic counselling.

Experiencia en el uso de terapia biológica en pacientes con infección por el virus de la inmunodeficiencia humana y psoriasis

IntroductionCutaneous pathologies are frequent on VIH patients, it has been described that this group of people has a similar if not higher prevalence of psoriasis, and however, there is little evidence on the usage of biological medication on these patients. The objective of this study is to describe sociodemographic and clinical characteristics of the VIH psoriasis patients that are treated with biological therapy at high complexity hospital. Materials and methodsObservational descriptive cross-sectional study conducted in Bogota, Colombia between 2016 and 2021. The sociodemographic, clinical and treatment characteristics in patients with HIV and psoriasis on biological therapy treated at a reference center. ResultsTwenty-one patients were included, 15 (71.43%) male and 6 (28.57) female, with an average age of 42.66 years old (SD 10.23). They all presented plaque psoriasis. Out of all patients, 52% had comorbidities of metabolic origin. The first-line biological treatment used the most was Ustekinumab (33%), Adalimumab (23.81%) and Etanercept (23.81%) followed by Secukinumab (9.52%), Ixekizumab (4.76%) and Guselkumab (4.76%); 52% didn’t register any infectious event. A statistically significant difference was evidenced on the PASI scores before and after 6 months of treatment (t = 12.05, p < 0.000). Similarly, improvement in CD4 count after first and second-line biologic therapy, regardless of mechanism of action. ConclusionsThis is the first study in our country that describes the usage of biological medication on VIH-Psoriasis patients, revealing the decrease on the PASI and increase on the CD4 count alongside antiretroviral medication.

Evaluating Real-World Characteristics of Patients With Advanced Urothelial Carcinoma Eligible for Avelumab Maintenance Therapy: A Multicountry Retrospective Medical Chart Review.

Urothelial carcinoma (UC) is a malignancy of the urothelium that encompasses the renal pelvis, bladder, and urethra. Current treatment guidelines for advanced (ie, locally advanced or metastatic) UC recommend using avelumab maintenance therapy in patients with nonprogressive disease following first-line platinum-based chemotherapy. This study aimed to assess the representativeness of the patient population in the JAVELIN Bladder 100 (JB-100) trial, which examined the efficacy and safety of avelumab first-line maintenance, vs. real-world patients with advanced UC that had not progressed with first-line platinum-based chemotherapy treated between 2015 and 2018 by reviewing demographic and clinical characteristics. A medical chart review (MCR) study collected demographics and treatment characteristics for patients with advanced UC in the United States, the United Kingdom, and France. Data were analyzed descriptively for review with data collected from patients enrolled in JB-100. Clinical characteristics were consistent between JB-100 and the MCR. Most patients were male, received 4 to 6 cycles of platinum-based chemotherapy, and had an Eastern Cooperative Oncology Group performance status of 0 or 1. All patients in the MCR had either stable disease or a response with platinum-based chemotherapy (∼75% achieved a complete or partial response). Fewer than half (42.5%) of all patients in the MCR received subsequent therapy. Patient demographics, clinical characteristics, and treatment patterns from a MCR of patients with advanced UC that had not progressed following first-line platinum-based chemotherapy appeared similar to data from patients enrolled in JB-100. Future studies should examine whether real-world outcomes are consistent with findings from JB-100. NCT02603432.

Clinical Outcomes of Patients with Metastatic Breast Cancer Treated with Hypo-Fractionated Liver Radiotherapy.

To retrospectively review the clinical outcomes of patients with metastatic breast cancer (MBCa) following liver directed ablative intent radiotherapy (RT). Demographics, disease and treatment characteristics of patients with MBCa who received liver metastasis (LM) directed ablative RT between 2004-2020 were analysed. The primary outcome was local control (LC), secondary outcomes included overall survival (OS) and progression-free survival (PFS) analyzed by univariate (UVA) and multi-variable analysis (MVA). Thirty MBCa patients with 50 LM treated with 5-10 fraction RT were identified. Median follow-up was 14.6 (range 0.9-156.2) months. Class of metastatic disease was described as induced (12 patients, 40%), repeat (15 patients, 50%) and de novo (three patients, 10%). Median size of treated LM was 3.1 cm (range 1-8.8 cm) and median biologically effective dose delivered was 122 (Q1-Q3; 98-174) Gy3. One-year LC rate was 100%. One year and two-year survival was 89% and 63%, respectively, with size of treated LM predictive of OS (HR 1.35, p = 0.023) on UVA. Patients with induced OMD had a significantly higher rate of progression (HR 4.77, p = 0.01) on UVA, trending to significance on MVA (HR 3.23, p = 0.051). Hypo-fractionated ablative liver RT in patients with MBCa provides safe, tolerable treatment with excellent LC.

Total CroFab and Anavip Antivenom Vial Administration in US Rattlesnake Envenomations: 2019–2021

IntroductionIn 2018, Anavip became available for the treatment of rattlesnake envenomations in the USA. No comparisons between the treatment characteristics of patients have been made since Anavip and CroFab have both been widely available. The objective of this study was to compare the number of antivenom vials administered of CroFab and Anavip during the treatment of rattlesnake envenomations in the USA.MethodsThis was a secondary analysis of rattlesnake envenomations utilizing the North American Snakebite Registry (NASBR) from 2019 through 2021. Frequencies and proportions were used to summarize demographics and baseline clinical characteristics. The primary outcome was total antivenom vials administered during treatment. Secondary outcomes included the number antivenom administration events, total treatment time, and hospital length of stay.ResultsTwo hundred ninety-one rattlesnake envenomations were analyzed; most occurred in the Western USA (n = 279, 96 %). One hundred one patients (35%) received only CroFab, 110 (38%) received Anavip only, and 80 (27%) received both products. The median number of vials used was 10 for CroFab, 18 for Anavip, and 20 for both antivenoms. More than one antivenom administration was necessary in thirty-nine (39%) patients that received only CroFab and 76 (69%) patients that received Anavip only. The median total treatment time was 5.5 hours for CroFab, 6.5 for Anavip, and 15.5 hours when both antivenoms were administered. All antivenom groups had a median hospital length of stay of 2 days.ConclusionsRattlesnake envenomated patients in the Western USA treated with CroFab had fewer antivenom vials and fewer antivenom administrations compared to patients treated with Anavip.

Recurring Fatigue After Biologic Administration: Patient-Reported Data from the Dutch Biologic Monitor.

Fatigue is a common problem in immune-mediated inflammatory disease (IMID) patients, significantly impacting their quality of life. In this study, we describe the pattern and characteristics of fatigue as a patient-reported adverse drug reaction (ADR) of biologics, and compared patient and treatment characteristics with patients reporting other ADRs or no ADRs. In this cohort event monitoring study, the description and characteristics of fatigue reported as a possible ADR in the Dutch Biologic Monitor were assessed and analysed for commonly recurring themes or patterns. Baseline and treatment characteristics of patients with fatigue and patients reporting other ADRs or no ADRs were compared. Of 1382 participating patients, 108 patients (8%) reported fatigue as an ADR of a biologic. Almost half of these patients (50 patients, 46%) described episodes of fatigue during or shortly after biologic injection, which often recurred following subsequent injections. Patients with fatigue were significantly younger than patients with other ADRs or patients without ADRs (median age for patients with fatigue, 52years; median age for patients with other ADRs, 56years; and median age for patients without ADRs, 58years); significantly more often smoked (25% vs. 16% and 15%); used infliximab (22% vs. 9% and 13%), rituximab (9% vs. 3% and 1%) or vedolizumab (6% vs. 2% and 1%); and significantly more often had Crohn's disease (28% vs. 13% and 13%) and other comorbidities (31% vs. 20% and 15%). Patients with fatigue significantly less frequently used etanercept (12% vs. 29% and 34%) or had rheumatoid arthritis (30% vs. 45% and 43%). IMID patients may experience fatigue as a postdosing effect of biologics.

Idecabtagene Vicleucel Chimeric Antigen Receptor T-Cell Therapy for Relapsed/Refractory Multiple Myeloma with Renal Insufficiency: Real World Experience

Introduction: Idecabtagene vicleucel (ide-cel) is an autologous BCMA directed chimeric antigen receptor therapy T (CAR-T) cell therapy approved for relapsed/refractory multiple myeloma (RRMM). However, patients with renal insufficiency (RI) were excluded from the registrational KarMMa clinical trial. RI impacts a significant proportion of patients with multiple myeloma and the safety and efficacy profile of CAR-T remains unknown in this patient population. We evaluated the real-world outcomes of patients with RI treated with standard of care ide-cel. Methods: RI was defined as creatinine clearance (CrCl) < 50 ml/min at the time of CAR-T therapy. CrCl of < 30 ml/min or being on dialysis was defined as severe RI. Dosing of lymphodepletion chemotherapy and grading/management of toxicities was per institutional guidelines, while responses were graded based on the IMWG response criteria. Results: The study cohort includes 211 patients receiving ide-cel from 11 medical centers, of which 28 (13%) patients had RI at the time of CAR-T therapy with CrCl < 50 ml/min. Among these, 11 (39%) patients had severe RI including one patient on dialysis. Table 1 describes baseline and treatment characteristics of patients with and without RI. Patients with RI were older (69 vs 63 years, p=0.003) and more likely to be female (68% vs 35%, p=0.001). They had lower albumin, higher beta-2-microglobulin (B2M) and higher likelihood of having revised ISS stage 3 disease, driven primarily by high B2M levels. Median prior lines of therapy in patients with and without RI was 8 vs 6, respectively. Fludarabine was dose reduced in 82% patients with RI and amongst this group 61% of patients had > 20% dose reduction. There was no difference in CAR-T cell dose infused (median: 416 vs 408 million cells). Cytokine release syndrome (CRS) was seen in 89% vs 84%, p=0.7 of patients with and without RI, including grade ≥ 2 CRS in 14% vs 20%, p=0.6 of patients, respectively. Neurotoxicity was observed in 21% vs 19%, p=0.7 of patients, respectively. Patients with RI had a longer hospital stay (median 13 vs 9 days, p=0.05), although ICU admission rates were similar (14% vs 7%, p=0.3). Infections were seen in 43% vs 32%, p=0.2 of patients, respectively. At one month, any ≥ grade 3 cytopenia was more common in patients with RI (86% vs 55%, p=0.001), including grade ≥ 3 neutropenia (54% vs 33%, p=0.04) and grade ≥ 3 thrombocytopenia (75% vs 41%, p<0.001). By 90 days post CAR-T, there was no difference in > grade 3 cytopenias amongst the 2 groups. Renal function did not significantly change after CAR-T in most patients. Amongst patients with paired baseline and day 30 data, no patient with CrCl 30-49 ml/min experienced worsening of CrCl to < 30 ml/min at day 30. Amongst 10 patients with CrCl < 30 ml/min, 3 patients experienced improvement in CrCl to 30-49 ml/min, while 7 patients had similar CrCl at day 30. New need for dialysis post CAR-T was observed in two patients, both with baseline CrCl > 50 ml/min; in one patient at 1 week post CAR-T in the setting of critical illness/severe CRS and in another patient 8 months post CAR-T in the setting of salvage therapy for relapse and critical illness. 34 patients have died by last follow-up, of which 8 were due to CAR-T related toxicities including infections (1 patient with RI, 7 without RI, p=0.9). 189 patients were evaluable for response. Patients with RI had an overall response rate of 96% comparted to 83% in patients without RI, p=0.045. Complete response (58% vs 65%, p=0.1) and very good partial response rates (77% vs 65%, p=0.2) were similar. At a median follow-up of 6 months, the median progression-free survival (PFS) in the two groups was 6.5 vs 8.1 months, p=0.6 (Fig 1). On multivariable analysis incorporating RI, age, cytogenetics and prior BCMA-directed therapy, RI was not an independent predictor for PFS (HR 1.4, 95% CI: 0.7-2.7, p=0.4), while high-risk disease, prior BCMA therapy, and age < 65 years were independent adverse prognostic factors. Conclusions: This multicenter retrospective study supports the feasibility of ide-cel CAR-T cell therapy in RRMM patients with RI. CRS, neurotoxicity and non-relapse mortality were comparable in patients with or without RI, although patients with RI had a longer hospital stay and were more likely to experience short-term high-grade cytopenias. Importantly, patients with RI experienced similar benefit in terms of response rates and PFS. S.S & L.P: co-first authors. L.S, K.K.P & D.K.H: joint senior authors Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Treatment Patterns of Real-World Patients with TRK Fusion Cancer Treated by US Community Oncologists.

Background Neurotrophic tyrosine receptor kinase (NTRK) gene fusions are present across various tumor types with an estimated overall prevalence of less than 1%. Tropomyosin receptor kinase inhibitors (TRKis) block the constitutively activated tyrosine receptor kinase (TRK) fusion protein produced in cancers with NTRK gene fusions (NTRK+) from downstream signaling. Many treatment guidelines now include TRKis as first-line (1L) or subsequent treatment options for TRK fusion cancer.ObjectiveThis study aimed to assess treatment patterns subsequent to a finding of NTRK+ status among patients with TRK fusion cancer.Patients and MethodsThis was a one-time, retrospective, multi-site patient chart abstraction by oncology practices in the USA from June to September 2020. US medical oncologists from the Oncology Provider Extended Network (OPEN) who had treated patients with NTRK+ advanced/metastatic solid tumors abstracted information into electronic case report forms (eCRFs) for adult patients with advanced/metastatic solid tumors and a NTRK+ tumor test result with a known fusion partner. Data abstracted into eCRFs by oncologists included demographic, clinical, and treatment characteristics of patients with advanced/metastatic TRK fusion solid tumors. Responses were summarized using descriptive statistics. Median treatment durations across the lines of therapy were estimated by Kaplan-Meier time to discontinuation.Results A total of 19 medical oncologists abstracted data from 110 patient charts.Median patient age at advanced/metastatic diagnosis was 62 years. The majority of patients were male (58.2%) and White (79.1%). Solid tumor types reported in at least 10% of the study cohort were lung (24.5%), cholangiocarcinoma (13.6%), pancreatic (10.9%), and colorectal (10.0%). Results for patients with hepatobiliary cancers (i.e., cholangiocarcinoma, pancreatic cancer, hepatocellular carcinoma) and colorectal cancer, and appendiceal cancer are also included. Median duration of 1L TRKi therapy was 16.8 months across all solid tumor types, whereas median duration of 1L was 5.6 months among patients receiving non-TRKi therapies (p = 0.017). Among the solid tumor types represented by at least 10% of the study population, median duration of 1L TRKi therapy was only reached in patients with pancreatic cancer (3.3 months). Median duration of TRKi in the second-line (2L) setting was 7.9 months overall, relative to 5.3 months among patients receiving non-TRKi therapies (p = 0.003). Across lung, cholangiocarcinoma, pancreatic, and colorectal cancers, the median durations of 2L TRKi therapy were 14.1, 6.0, 6.1, and 4.1 months, respectively.Conclusion and RelevanceAmong patients with advanced/metastatic TRK fusion solid tumors, medical oncologists reported that approximately two-thirds initiated a TRKi during the study period. Treatment with a TRKi was longer in duration compared to non-TRKi treatment in 1L and 2L therapy. Additional research is needed to gain insight into the association between early TRKi therapy initiation and clinical outcomes in the real-world setting.Supplementary InformationThe online version contains supplementary material available at 10.1007/s11523-022-00909-7.

Costs of acute hospitalisation for stroke and transient ischaemic attack in Australia.

Stroke is a high-cost condition. Detailed patient-level assessments of the costs of care received and outcomes achieved provide useful information for organisation and optimisation of the health system. To describe the costs of hospital care for stroke and transient ischaemic attack (TIA) and investigate factors associated with costs. Retrospective cohort study using data from the Australian Stroke Clinical Registry (AuSCR) collected between 2009 and 2013 linked to hospital administrative data and clinical costing data in Queensland. Clinical costing data include standardised assignment of costs from hospitals that contribute to the National Hospital Costing programme. Patient-level costs for each hospital admission were described according to the demographic, clinical and treatment characteristics of patients. Multivariable median regression with clustering by hospital was used to determine factors associated with greater costs. Among 22 hospitals, clinical costing data were available for 3909 of 5522 patient admissions in the AuSCR (71%). Compared to those without clinical costing data, patients with clinical costing data were more often aged <65years (30% with cost data vs 24% without cost data, p < 0.001) and male (56% with cost data vs 49% without cost data, p < 0.001). Median cost of an acute episode was $7945 (interquartile range $4176 to $14970) and the median length of stay was 5days (interquartile range 2 to 10days). The most expensive cost buckets were related to medical (n = 3897, median cost $1577), nursing (n = 3908, median cost $2478) and critical care (n = 434, median cost $3064). Factors associated with greater total costs were a diagnosis of intracerebral haemorrhage, greater socioeconomic position, in-hospital stroke and prior history of stroke. Medical and nursing costs were incurred by most patients admitted with stroke or TIA, and were relatively more expensive on average than other cost buckets such as imaging and allied health. Scaling this data linkage to national data collections may provide valuable insights into activity-based funding at public hospitals. Regular report of these costs should be encouraged to optimise economic evaluations.

P.139 Pediatric posterior fossa ependymoma recurrence in a molecularly defined cohort – Clinical, demographic, and surgical factors associated with outcome

Background: Pediatric posterior fossa ependymoma contributes to morbidity and mortality in children. Following gross total resection and adjuvant radiotherapy, there is a known risk of local recurrence that portends a dismal prognosis. We sought to characterize survival in a molecularly defined cohort with an emphasis on recurrence patterns that influence outcome. Methods: This study was approved by the Ethics Board of the Hospital for Sick Children. We performed a twenty-year single-center retrospective study to identify clinical, demographic and treatment characteristics of patients with pathologically diagnosed posterior fossa ependymoma. Results: There were 60 patients identified that underwent primary resection. Recurrence rate in the cohort was 48% with 29 cases of recurrent ependymoma occurring at a mean time of 24 months after index surgery. No mortalities were observed among patients undergoing primary resection without recurrent disease. Median cohort survival was 12.3 years in the primary cohort and and 6.32 years among patients recurrent ependymoma. Recurrent disease was significantly associated with worse overall survival after multivariate analysis (HR = 0.024). Conclusions: We highlight overall survival and factors influencing mortality in pediatric posterior fossa ependymoma. Recurrent disease confers a worse prognosis. We describe for the first time survival trends following local and distant recurrences managed through multiple resections.

Racial and ethnic differences in presentation and clinical outcomes for pediatric rhabdomyosarcoma (RMS).

10017 Background: Race and ethnicity are recognized risk factors for many cancer types, including several pediatric cancers. Though racial and ethnic disparities in the presentation, treatment, and survival of certain cancers have been widely demonstrated, there is conflicting evidence about whether such disparities exist in RMS, the most common pediatric soft tissue sarcoma. Understanding the role of race and ethnicity in the presentation, treatment, and prognosis of RMS is important to promote improved survival in patients of all racial and ethnic groups. Methods: Patient, tumor, and treatment characteristics of patients enrolled on Children’s Oncology Group studies D9602, D9802, D9803, ARST0331, ARST0431, ARST0531, and ARST08P1 were compared across racial and ethnic groups using a chi-square test. Significant characteristics underwent pairwise analysis, comparing the Non-Hispanic Black (NHB) and Non-Hispanic White (NHW) groups. Outcome analyses were performed using the Kaplan-Meier method and Wilcoxon signed-rank test. Results: Race and ethnicity incidence among the 2157 study patients were as follows: 8 (0.4%) American Indian or Alaska Native, 56 (2.6%) Asian, 271 (12.6%) Hispanic, 4 (0.2%) Native American or other Pacific Islander, 275 (12.8%) Non-Hispanic Black, 1335 (61.9%) Non-Hispanic White, and 208 (9.6%) unknown. Thirteen patient and tumor factors relating to presentation and treatment were evaluated for differences by race and ethnicity; the following five were significant: age, IRS group, tumor invasiveness, metastatic disease, and FOXO1 fusion partner. Pairwise comparison of NHB and NHW patients for these factors demonstrated that NHBs are more likely than NHWs to present at age 10 years or greater (p = 0.002) and that NHB patients are more likely to present with invasive tumors (p = 0.012). Incidence of metastatic disease at diagnosis was not significantly different between the groups (p = 0.202). No differences in treatment, including extent of surgical resection (p = 0.259) or use of radiation therapy (p = 0.920), were found. Neither event free survival nor overall survival were significantly different across the entire cohort (EFS p = 0.457, OS p = 0.159) or in subset analysis by risk group (low risk: EFS p = 0.856, OS p = 0.558; intermediate risk: EFS p = 0.907, OS p = 0.493; high risk: EFS p = 0.218, OS p = 0.397), by age (&lt; 1y: EFS p = 0.489, OS p = 0.546; 1-9y: EFS p = 0.417, OS p = 0.112; ≥10y: EFS p = 0.556, OS p = 0.609), in invasive tumors (EFS p = 0.704, OS p = 0.872), or in metastatic disease (EFS p = 0.270, OS p = 0.373). Conclusions: These data indicate that while differences in the presenting features of RMS exist between racial groups, with NHB patients exhibiting higher risk features, patients treated on these clinical trials did not experience differences in outcomes by racial group. This suggests NHB patients may experience a survival benefit from clinical trial enrollment.

Profile of Patients with Diabetes Treated with Insulin Lispro 200 U/ml: A Real-World Study from Spain.

IntroductionInsulin lispro 200 U/ml (IL200) is a rapid-acting concentrated insulin used for the treatment of adults with diabetes requiring daily doses of > 20 units of rapid-acting insulin. The aim of this study was to describe the clinical/demographic and treatment characteristics of patients who initiated insulin IL200 therapy in Spain in a real-world setting (PROFILE-IL200).MethodsThis retrospective observational study based on the IQVIA database included adult (≥ 18 years) patients with type 1 (T1D) or type 2 (T2D) diabetes who initiated IL200 between June 2015 and December 2019. Demographic and clinical characteristics were analyzed descriptively.ResultsMain characteristics for the T1D/T2D groups (N = 65/167) were as follows: male, 63.1/55.7%; mean (standard deviation [SD]) age, 46.5 (15.5)/62.6 (12.8) years; time since first diabetes record, 6.6 (4.2)/7.9 (2.9) years; body mass index (BMI), 30.9 (5.8)/33.1 (5.5) kg/m2; glycated hemoglobin, 8.3 (2.1)/8.8 (1.8)%; and diabetes-associated comorbidity, 55.4/92.8%. Among patients with T1D/T2D and a prior diagnosis (N = 54/164), 96.3/90.2% had received previous insulin (rapid insulin in 81.5/62.2%), and 13.0/97.6% had received previous noninsulin antihyperglycemic therapy. The mean (SD) total insulin dose before IL200 initiation for T1D/T2D was 98.0 (73.9)/95.2 (59.8) U/day; IL200 was initiated at a dose of 56.3 (43.8)/51.5 (34.3) U/day, with basal insulin in 86.2/83.2% of the patients. IL200 was first prescribed by an endocrinologist or a primary care physician in 48.7% and 46.6% of patients, respectively.ConclusionsPROFILE-IL200 described the profile of patients treated with IL200 in clinical practice in Spain. Patients were middle-aged, with poor glycemic control, high BMI and associated comorbidities, and received high doses of insulin at IL200 initiation.Supplementary InformationThe online version contains supplementary material available at 10.1007/s13300-022-01264-6.

Demographic, clinical, laboratory data, prognostic, and treatment features of patients with antisynthetase syndrome: An international, two-center cohort study.

To compare clinical, demographic, laboratory data, prognostic and treatment characteristics of patients with antisynthetase syndrome (ASSD) treated in two different centers of India and Brazil. This international, two-center, retro-prospective cohort study which was conducted at two tertiary rheumatology centers (one in Brazil and one in India) between January 2000 to January 2020 included a total of 115 patients with ASSD (21 males, 94 females; mean age; at disease diagnosis at 40.3; range, 18 to 80 years). Demographic, clinical and laboratory data of the patients were recorded. Clinical involvement was evaluated. Of the patients, 81 were Brazilians and 34 were of Indian origin. The Indian group exhibited a greater delay in diagnosis after the onset of symptoms compared to Brazilian patients (12 vs. 6 months, respectively; p=0.026). Brazilian patients exhibited a significantly higher prevalence of joint and lung involvement, mechanic's hands, and Raynaud's phenomenon. Anti-Jo-1 was the most common autoantibodies in both groups. Systemic arterial hypertension, followed by diabetes mellitus were the most prevalent comorbidities. Concerning previously used drugs, the Indian patients had a larger group of patients treated with antimalarials, whereas the Brazilian group used more azathioprine and intravenous immunoglobulin. A higher proportion of Indian patients was treated with one immunosuppressive drug (70.6%), while the Brazilian group were often treated using two immunosuppressive drugs (33%). Comparison between the severity and prognosis showed that Brazilian group had a higher number of relapses, and during follow-up, the global mortality rates were similar in both groups (6.2% for Brazilian vs. 8.8% for Indian). Brazilian and Indian patients with ASSD have comparable epidemiological characteristics such as age at the time of disease diagnosis, and sex distribution, and autoantibodies. Diagnostic delay is seen in Indian patients, and Brazilians exhibit a higher prevalence of joint and lung involvement, mechanic's hands, Raynaud's phenomenon with a higher number of relapses, although the mortality rate seems to be similar in both groups.

POSB393 Profile of Patients with Type 1 or Type 2 Diabetes Treated with Insulin Lispro 100 Units/ML Junior Kwikpen in Routine Clinical Practice in Spain

Insulin lispro 100 units /mL Junior KwikPen (JrKwikPen) is a disposable pre-filled pen that delivers 0.5–30 units (U) in increments of 0.5 U, which allows for more accurate insulin dose adjustment and more precise glycaemic control. This study describes sociodemographic, clinical, and treatment characteristics of patients with diabetes who initiated treatment with JrKwikPen in real world practice in Spain. This observational retrospective study, based on IQVIA’s electronic medical records databases, included patients of all ages with type 1 diabetes (T1D) or type 2 diabetes (T2D) who started treatment with JrKwikPen between 1st May 2018 and 31st December 2020. Patient characteristics and concomitant treatment at JrKwikPen initiation (index date) and prior medical/treatment history were analysed descriptively by type of diabetes and age groups. A total of 416 patients were included. Main characteristics for T1D/T2D groups (N=326/90) were, respectively: female, 61.7%/65.6%; mean age (standard deviation [SD]), 32.5 (20.7)/55.5 (16.6) years; time since first diabetes record, 5.8 (5.1)/7.9 (4.2) years; body mass index (BMI), 20.8 (4.2)/24.8 (4.8) kg/m2 (N=106/44); haemoglobin A1c (HbA1c), 8.2 (2.0)%/8.0 (1.4)% (N=67/38); presence of diabetes-associated comorbidities, 27.9%/64.4%. Only 32.8% of patients with T1D were <18 years. Among JrKwikPen users 12.3% (T1D/T2D, 6%/6.3%) were ≥65 years, 20.9% patients were newly diagnosed, and 3.8% were pregnant women. The mean (SD) total insulin dose pre-index for T1D/T2D was 42.4 (23.4)/40.7 (21.6) U/day, respectively. The mean (SD) insulin dose at initiation of JrKwikPen was 26.6 (16.6) and 22.6 (13.6) U/day for T1D/T2D, respectively. JrKwikPen was first prescribed mainly by endocrinologists (58.7%) or pediatricians (22.6%). This is the first study that describes the profile of patients treated with insulin lispro 100 U/mL JrKwikPen in clinical practice in Spain. JrKwikPen users were mainly women, adults, with T1D, low BMI, and more associated comorbidities.

Effects of Adherence to Pharmacological Treatment on the Recovery of Patients with Schizophrenia.

The aim of this study was to evaluate the effects of adherence to antipsychotic treatment on the recovery of patients with schizophrenia in northern Chile. One hundred and fifty-one patients diagnosed with schizophrenia completed the Drug Attitude Inventory (DAI-10), Positive and Negative Syndrome Scale for Schizophrenia (PANSS), Recovery Assessment Scale (RAS-24), sociodemographic information, and clinical and treatment characteristics of patients with schizophrenia. Multivariate analysis with multiple linear regression was then performed to identify variables that were potentially associated with the recovery assessment (variable criterion). A significant association was found between adherence to antipsychotic medication and the Willing to Ask for Help dimension of Recovery (β = 0.239, p = 0.005). Association of clinical and socio-demographic variables with recovery were identified: negative symptoms with Personal Confidence and Hope (β = −0.341, p = 0.001) and Goal and Success Orientation (β = −0.266, p = 0.014); cognitive symptoms with Willing to Ask for Help (β = −0.305, p = 0.018) and no domination by symptoms (β = −0.351, p = 0.005); marital status with reliance on others (β = −0.181, p = 0.045); age with Personal Confidence and Hope (β = −0.217, p = 0.021), Goal and Success Orientation (β = −0.296, p = 0.003), and no domination by symptoms (β = 0.214, p = 0.025). Adherence has a positive relationship with personal recovery in this sample of Chilean patients with schizophrenia.