Aggressive Therapy Research Articles

Impella support for refractory cardiogenic shock accompanied by diabetic ketoacidosis: a case report.

Sodium-glucose cotransporter 2 (SGLT2) inhibitors are strongly recommended in patients with heart failure, regardless of the presence of diabetes mellitus. A 74year-old woman with a reduced left ventricular ejection fraction and diabetes mellitus (the types were unknown), receiving insulin and SGLT2 inhibitor, was hospitalized for altered consciousness with systemic hypotension. Upon admission, she was diagnosed with cardiogenic shock due to diabetic ketoacidosis. Intensive fluid resuscitation under Impella CP support successively improved her metabolic acidosis, preventing worsening pulmonary congestion by mechanically unloading the heart. After hemodynamic stabilization, she was diagnosed with type 1 diabetes mellitus for the first time. She was discharged on day 54 and was followed for 6months without any recurrences. We must remain vigilant regarding the risk of diabetic ketoacidosis in patients using SGLT2 inhibitors, particularly those on insulin therapy or with diabetes mellitus of unknown types. Impella device shows promise as a circulatory support system in alleviating the left ventricle's workload and averting exacerbated pulmonary congestion, especially in cases where patients necessitate aggressive fluid replacement therapy, such as in the treatment of diabetic ketoacidosis concurrent with compromised cardiac function.

Combination therapy for hypertension management: insights from the Indian experts

Cardiovascular diseases (CVDs) pose a significant global health challenge, with India bearing a disproportionate burden of CVD-related morbidity and mortality. Hypertension (HTN) is a major risk factor for CVDs, affecting nearly 30% of the Indian population. Achieving target blood pressure (BP) levels is crucial for reducing cardiovascular risk, necessitating aggressive antihypertensive therapy. Combination therapy has emerged as a cornerstone in HTN management, especially in high-risk patients. This review delves into the literature and perspectives of Indian cardiologists on combination therapy for HTN management. Despite the efficacy of contemporary antihypertensive medications, a substantial proportion of patients fail to reach target BP levels with monotherapy. Combination therapy offers synergistic effects, addressing multiple pathways involved in HTN pathogenesis. Recent guidelines recommend initiating treatment with two-drug combinations, transitioning to three-drug combinations in resistant cases. Combination therapy not only enhances BP control but also reduces the risk of cardiovascular events and mortality compared to monotherapy. Optimal management of HTN requires personalized approaches, considering individual patient profiles and comorbidities such as coronary artery disease (CAD), diabetes mellitus (DM), dyslipidemia, and heart failure (HF). In such cases, combination therapy plays a pivotal role in mitigating cardiovascular risks. ARB/CCB combination therapy, particularly telmisartan/amlodipine, demonstrates significant efficacy and tolerability across various patient populations, including those with metabolic risk factors and renal impairment. Expert recommendations highlight the importance of individualized therapy, patient education, early diagnosis, and initiation with dual therapy in India. Strategies to improve medication adherence and compliance, such as single-pill double or triple combinations, are emphasized. Moreover, awareness of newer treatment options and contactless diagnostic instruments is crucial for optimizing HTN management. In conclusion, combination therapy stands as a cornerstone in HTN management, offering enhanced efficacy, tolerability, and cardiovascular protection. Tailored approaches guided by expert recommendations are essential to address the growing burden of HTN and reduce the socioeconomic impact of CVDs in India.

#2977 Pregnancy-related acute kidney injury in a second level care hospital in Merida, Yucatan, Mexico

Abstract Background and Aims Pregnancy-related acute kidney injury (PRAKI) is a public health problem associated with an increased risk of adverse maternal and fetal outcomes. The causes of PRAKI varies according to the age of gestation. Mexico lacks a national acute kidney injury (AKI) registry, and the access to AKI treatment is limited due to lack of financial coverage, lack of nephrologists, and poor infrastructure. The General Hospital Dr. Agustín O'Horán is a second level unit that attends to the uninsured pregnant population from the southeast of Mexico. The aim of this study was to investigate the prevalence, clinical characteristics, and outcomes of PRAKI in a Mexican population. Method This was a retrospective study in an obstetric intensive care unit (OICU) in the General Hospital Dr. Agustín O'Horán in Merida, Yucatan, Mexico. Women during pregnancy and postpartum periods admitted to the OICU between January to December 2023 were included. Demographical, clinical and laboratory data were collected from medical records, and AKI was diagnosed according to the Kidney Disease Improving Global Outcomes (KDIGO) creatinine criteria. We evaluated the prevalence, clinical characteristics, and outcomes of PRAKI. Results During the study period, 188 women were admitted to the OICU, and the mean age was 24.9 years. Eight women presented in the first trimester, 19 in the second trimester, 21 in the third trimester, and 140 in the postpartum period. Fifty-five women met AKI creatinine criteria: 31 (56.3%) had stage 1, 15 (27.3%) had stage 2, and 9 (16.4%) had stage 3. The most common causes of PRAKI were obstetric hemorrhage (27.3%), eclampsia (21.8%), and preeclampsia (18.2%) (Table 1). There was no difference in vital signs on admission and baseline creatinine level between both groups. Women who developed PRAKI had a longer stay in the OICU (4.2 days vs 2.3 days, p < 0.001), and greater use of vasopressor (30.9% vs 9.0%, p < 0.001), and mechanical ventilation (34.5% vs 11.3%, p < 0.001). Women with AKI had higher mortality (9.1% vs 1.5%, p = 0.012), and perinatal mortality (9.1% vs 2.3%, p = 0.035) (Table 2). Conclusion Women during pregnancy and postpartum periods who developed PRAKI had more adverse clinical and obstetric outcomes (maternal and perinatal mortality). Hence, women in pregnancy and postpartum periods admitted to an intensive care unit should be closely monitored and implement strategies for early diagnosis and aggressive therapy of PRAKI regardless of KDIGO stage.

Survival and predictors of adult patients admitted to intensive care unit in Yekatit 12 Hospitals, Addis Ababa, Ethiopia.

Background In hospitals, one of the main service components is an intensive care unit (ICU) that provides aggressive therapy for critically ill and high-risk patients. The availability of ICU beds has been progressively growing in Africa, but many studies show that the ICU mortality rate is very high. However, many of those studies are only descriptive and focus on medical or surgical patients. This study includes patients from all wards except pediatrics. Methods A retrospective cohort study was carried out in the intensive care unit at Yekatit 12 Hospital, Ethiopia. The Kaplan-Meier method was used to describe the probability of survival in ICU stay. The Cox proportional hazard model was used for the multivariate analysis to determine the possible associations of predictor variables and to obtain the adjusted hazard ratios. A statistically significant association was declared at p <0.05 with a 95% confidence interval. Results The survival rate was 69.7% with a mortality rate of 30.3%. This study confirmed that the risk of mortality among ICU patients was the education status of the study participants, attending primary education was twice that of patients attending higher education with an adjusted hazard ratio (AHR) (AHR=2.097, 95% CI:1.081,4.067). Patients admitted to ICU because of shock were more than four times at risk of death compared to other causes (AHR= 4.51, 95% CI: 2.41, 8.45). The risk of mortality among ICU patients admitted because of brain injury was more than two times compared to other patients (AHR=2.77, 95% CI: 1.18, 6.47). Patients with respiratory failure were more than two times at risk of mortality (AHR=2.42, 95% CI: 1.19, 4.87). Conclusions The survival of ICU patients was low. Formal education level, patients admitted for shock, brain injury, and respiratory failure were found to be significantly associated with the survival of ICU patients.

A NEGLECTED RARE DISORDER: IGG4 RELATED DISEASE

Abstract IgG4–related disease (IgG4–RD) is an autoimmune disorder characterized by infiltration of plasma cells with IgG4. Almost any organ could be affected. Common presentations include lung manifestation, autoimmune pancreatitis, retroperitoneal fibrosis and tubulointerstitial nephritis. Cardiovascular involvement is less frequent. Corticosteroids are first line therapy, with or without anti–rheumatic drugs. Long–term prognosis of IgG4–RLD remains unknown. We present the case of a 65–years old woman, with a previuos diagnosis of IgG4–RD with mediastinal involvement. In the meantime she was recovered for pyelonephritis with high fever and positive blood coltures (S. hominis). We were called to perform a transthoracic echocardiogram to rule out infective endocarditis. The exam showed normal size of cardiac chambers, normal biventricular systolic function, absence of relevant valvular disease. In apical view we noticed a hyperechogenic ovalar imagine in the right atrium, not visible in all the projections. A first differential diagnosis included a mass with site of implantation at the top of the right atrium or something external but contiguous to heart structures. To clear this doubt the patient performed a thoracic CT with stunning results. The CT highlighted parietal encasement of chardiac structures and, particularly, of the superior vena cava at the confluence in right atrium, explaining the image we saw with transthoracic echo. The exam also showed encasement of ascending aorta, aortic arch and descending aorta with maximum thickness of 18 mm. CT also reported encasement of right pulmonary arthery. The CT highlighted a progression of the disease with indication to more aggressive therapy. In fact IgG4–RD of the cardiovascular system results in serious complications that need to be recognized and treated. Aortic involvement in IgG4–RD is extremely rare and tipically affects the infrarenal abdominal aorta. Cardiovascular IgG4–RD could also lead to aortitis, medium–vessel arteritis, pulmonary vascular disease, phlebitis, valvulopathy, pericarditis and myocardial disease. These forms of Igg4–RD are often underdiagnosed as clinical presentations is similar to cardiovascular disease due to more common aetiologies. Regarding our case the patient began methotrexate based on the results of the CT. This was a really interesting case for our working group to deepen this little–known disease. “The eyes see only what the mind is prepared to comprehend”.

Acute Surgical and Rehabilitation Management of Complex Hand Burns in Combat Casualties

Burns are inevitable in modern warfare and have comprised between 5% and 20% of battlefield injuries. Involvement of the hands is the leading cause of postburn functional impairment. The purpose of this paper is to provide guidance on aspects of care necessary for the management of complex hand burns in a battlefield setting. Proper assessment and establishment of a comprehensive plan of care at the onset of injury help to ensure optimal functional outcomes in hand function. Basic treatment principles for the acutely burned hand include edema management; early wound coverage, including excision of the burn and skin grafting; early and aggressive hand therapy; and burn-scar contracture mitigation strategies.

Abstract PO5-06-03: Impact of curative-intent treatment on survival for metastatic breast cancer limited to distant lymph nodes

Abstract BACKGROUND: Metastatic breast cancer limited to distant lymph nodes is classified as stage IV but potentially represents an anatomically distinct entity amenable to curative treatment. Its optimal management is unclear, with existing studies limited by small sample sizes and short follow-up durations. We sought to investigate the impact of curative-intent treatment on breast cancer-specific survival (BCSS) and overall survival (OS) for patients with this diagnosis. METHODS: Three cohorts of patients diagnosed with breast cancer from 2010 to 2019 were identified from the Surveillance, Epidemiology, and End Results 17 registries database. The curative cohort consisted of patients with metastatic breast cancer involving only distant lymph nodes who received a combination of chemotherapy, surgery to the primary site, and radiotherapy (curative-intent treatment). The palliative cohort included patients who underwent chemotherapy but not curative-intent treatment. The N3c cohort was comprised of patients with ipsilateral supraclavicular lymph node metastases but no distant metastases who received curative-intent treatment. Weighting based on covariate balancing propensity scores was performed to balance age, sex, race, median household income, year of diagnosis, T stage, histology, pathological grade, and ER/PR/HER2 status between the cohorts. Weight-adjusted multivariable Cox regression models with the same covariates were then used to compare the BCSS and OS of the three cohorts. RESULTS: A total of 2,089 patients with a median follow-up of 37 months were eligible for analysis, of whom 577 (27.6%) were in the curative cohort, 905 (43.3%) were in the palliative cohort, and 607 (29.1%) were in the N3c cohort. After propensity score weighting, the maximal standardized mean difference for all covariates was less than 0.1. Five-year BCSS for patients in the weight-adjusted curative, palliative, and N3c cohorts were 63.0% (95% CI 58.1-68.3), 46.7% (42.4-51.4), and 66.4% (61.4-71.8), respectively; and five-year OS were 57.9% (53.0-63.2), 41.2% (37.2-45.7), and 62.2% (57.3-67.6), respectively. Compared to the palliative cohort, the curative cohort exhibited better BCSS (HR 0.47, 95% CI 0.38-0.57; P< 0.001) and OS (HR 0.48, 95% CI 0.40-0.58; P< 0.001). In contrast, the N3c cohort had similar BCSS (HR 0.93, 95% CI 0.73-1.17; P=0.53) and OS (HR 0.96, 95% CI 0.77-1.19; P=0.68) to the curative cohort. CONCLUSIONS: Patients treated curatively for metastatic breast cancer limited to distant lymph nodes had higher BCSS and OS than those receiving palliative chemotherapy and similar BCSS and OS to patients receiving curative-intent treatment for ipsilateral supraclavicular lymph node metastases. These results support the use of aggressive multimodal therapy for select stage IV breast cancer patients with only distant nodal metastases and, if confirmed, suggest that a substantial portion of such patients may be undertreated. Citation Format: Yang Xu. Impact of curative-intent treatment on survival for metastatic breast cancer limited to distant lymph nodes [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO5-06-03.

Abstract PS11-02: Clinical risk factors of Central Nervous System (CNS)-related death in patients with HER2-positive metastatic breast cancer

Abstract Background: Central nervous system (CNS) relapse represents a challenge in the management of patients with HER2-positive (+) metastatic breast cancer. Data regarding the impact of brain metastases (BrM) and CNS involvement on mortality are sparse. In this study we sought to determine the proportion of HER2+ MBC patients in which CNS disease is the cause of death and identify risk factors associated with CNS-related mortality. Methods: We reviewed medical records of 294 consecutive patients with HER2+ MBC and diagnosis of CNS disease including parenchymal BrM, leptomeningeal disease (LMD) or dural metastases (DM), treated at Memorial Sloan Kettering Cancer Center between August 2010 and April 2022. HER2-positivity was assessed at the first diagnosis of metastatic disease (any site). Clinicopathologic characteristics including disease burden at presentation, timing of CNS disease and extracranial metastases (ECM), neurologic complications, and local and systemic treatments were collected. CNS-related death was defined as any death caused by BrM or LMD or DM progression or CNS treatment-associated complications. CNS-related death was estimated using cumulative incidence in the competing risks setting (with death due to other causes as a competing event) and overall survival (OS) was estimated using Kaplan Meier methodology. Risk factors associated with CNS-related death were assessed using sub-distribution hazards regression modeling. Treatments given after CNS disease diagnosis were treated as time-varying variables. Tests were two-sided with statistical significance < 0.05. Results: After excluding 19 patients (11 for discordant HER2 status, 5 for concomitant second solid tumor, and 3 for missing data), 275 patients were included in these analyses (258 patients with parenchymal BrM +/- LMD, 8 patients with DM only, 2 patients with DM concomitantly with LMD, and 6 patients with LMD only). Overall, 63/275 (23%) presented with CNS as first and only site of metastasis, 210 (76%) patients developed CNS disease synchronously with or following ECM, and 2 had unknown timing. 125/275 (45%) had de novo MBC, and nearly all patients were treated with CNS local therapies (92% ≥1 radiation treatment and 28% underwent ≥1 BrM resection). The median number of lines of systemic therapy after CNS disease diagnosis was 1 (range: 0-14); 105/275 (38%) patients received a HER2 tyrosine kinase inhibitor (lapatinib: 21%, neratinib: 4.4%, and tucatinib: 12%). The median follow-up was 3.6 years for survivors (range: 0.22 years-12 years). 193/275 (70%) patients died, of whom 105 (54%) died of CNS-related cause. The 3-year OS rate was 40% (95% CI=34%-46%), which varied for patients with BrM only at the diagnosis of metastatic disease [56% (95% CI=43%-69%] compared to patients with ECM +/- BrM [35% (95% CI=28%-42%] (p=0.05). The cumulative incidence of CNS-related death at 3 years was 33% (95% CI=27%-39%). Upon univariable modeling, LMD and CNS radiation treatment (RT) were associated with CNS-related death, and these associations remained statistically significant in a multivariable model [LMD: HR=2.49 (95% CI=1.62-3.83), p< 0.0001, and RT: HR=2.91 (95% CI=1.27-6.64), p=0.01]. Conclusions: Greater than half of patients with HER2+ with CNS involvement suffered of CNS-related death, with greatest risk among patients with LMD. We hypothesize that the association between radiotherapy and CNS-related death may reflect upfront response to systemic cancer-directed treatments of those in patients with DM only or limited CNS disease extension. CNS-only relapse at metastatic presentation portended improved survival than intra- plus extracranial progression, supporting an approach of aggressive local therapy for selected patients. Multimodality approaches using HER2-directed agents effective against parenchymal BrM, LMD along with extracranial metastases, in combination with local CNS-directed therapies need to be optimized. Citation Format: Emanuela Ferraro, Rabih Bou Nassif, Anne Reiner, Samantha Brown, Umberto Tosi, Katherine Panageas, Chau Dang, Andrew D Seidman, Nelson Moss. Clinical risk factors of Central Nervous System (CNS)-related death in patients with HER2-positive metastatic breast cancer [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PS11-02.

Rate of Change in Cardiac Magnetic Resonance Imaging Measures Is Associated With Death in Duchenne Muscular Dystrophy.

Cardiovascular disease is the leading cause of death among patients with Duchenne muscular dystrophy (DMD). Identifying patients at risk of early death could allow for increased monitoring and more intensive therapy. Measures that associate with death could serve as surrogate outcomes in clinical trials. Duchenne muscular dystrophy subjects prospectively enrolled in observational studies were included. Models using generalized least squares were used to assess the difference of cardiac magnetic resonance measurements between deceased and alive subjects. A total of 63 participants underwent multiple cardiac magnetic resonance imaging and were included in the analyses. Twelve subjects (19.1%) died over a median follow-up of 5 years (interquartile range, 3.1-7.0). Rate of decline in left ventricular ejection fraction was faster in deceased than alive subjects (P<0.0001). Rate of increase in indexed left ventricular end-diastolic (P=0.0132) and systolic (P<0.0001) volumes were higher in deceased subjects. Faster worsening in midcircumferential strain was seen in deceased subjects (P=0.049) while no difference in global circumferential strain was seen. The rate of increase in late gadolinium enhancement, base T1, and mid T1 did not differ between groups. Duchenne muscular dystrophy death is associated with the rate of change in left ventricular ejection fraction, midcircumferential strain, and ventricular volumes. Aggressive medical therapy to decrease the rate of progression may improve the mortality rate in this population. A decrease in the rate of progression may serve as a valid surrogate outcome for therapeutic trials.

Risk of first recurrence after treatment in a population-based cohort of young women with breast cancer.

Breast cancer (BC) in women under 45 is rare yet often aggressive. We aim to analyze loco-regional recurrences (LR), distant recurrences (DR), second breast cancers, and mortality in young BC patients. We enrolled 776 women with non-metastatic BC ≤45years diagnosed from 1970 to 2012. Variables included age, family history, tumor stage/grade, and treatment. We used multivariate Cox regression and competing risk models. Among the participants, 37.0% were diagnosed before the age of 40. Most had stage I or II, grade II, ER- and PR-positive, HER2-negative tumors. Over a median follow-up of 8.7years, 10.1% experienced LR, 13.7% developed DR, and 10.8% died, primarily due to BC. The majority of recurrences occurred within the first five years. Older age (>40) significantly reduced the risk of LR and DR. Advanced disease stage, certain surgical strategies, and positive margins increased DR risk. In the cohort diagnosed between 2001 and 2012, recent diagnosis, triple-negative cancer, and hormonal therapy were associated with reduced LR risk. Breast-conserving surgery appeared to offer protective effects against DR. This study highlights that BC in young women carries a significant risk of early recurrence, with age, tumor characteristics, and treatment modalities influencing outcomes. The findings emphasize the need for tailored treatment strategies for young BC patients, focusing on surgical precision and aggressive adjuvant therapy for high-risk cases. This research contributes valuable insights into managing BC in younger patients, aiding in improving long-term outcomes.

Early and aggressive treatment for Crohn disease using biologics and immunomodulators.

Traditionally, medical providers have used the step-up approach to manage patients with Crohn disease, starting with 5-aminosalicylic acid derivatives, progressing to corticosteroids, and eventually to immunomodulators and biologics. However, a new top-down approach focuses on early and aggressive therapy with biologics and immunomodulators to reduce the rate of mucosal and intestinal damage. This article describes early and aggressive biologic and immunomodulator therapies and new therapeutic parameters compared with traditional step-up treatment for patients with Crohn disease.

Impact of ischemic stroke topography on early clinical outcome of basilar artery occlusion: a retrospective study.

Basilar artery occlusion (BAO) may be etiologically attributed to embolism or in situ thrombosis due to basilar stenosis (BS). Patients with BAO due to BS (BAOS) are known to have worse outcomes than patients with embolic occlusions (BAOE). BAOS occurs more proximally in the basilar artery (BA) than BAOE. We hypothesize that differing brain stem infarct patterns contribute to outcome differences between these stroke etiologies. This retrospective study includes 199 consecutive patients with BAO who received endovascular treatment at a single center. Final infarction in brain parenchyma dependent on the posterior circulation was graded semiquantitatively on magnetic resonance imaging (MRI). Associations to underlying stenosis and angiographic and clinical outcome variables were tested. The primary endpoint was early good clinical outcome (EGCO, mRS score ≤ 3 at discharge). Infarct extension of the medulla oblongata (OR = 0.25; 95% CI = 0.07-0.86; p = 0.03), the inferior pons (OR = 0.328; 95% CI = 0.17-0.63; p = 0.001), the superior pons (OR = 0.57; 95% CI = 0.33-0.99; p = 0.046), and the occipital lobes (OR = 0.46; 95% CI = 0.26-0.80; p = 0.006) negatively predicted EGCO. Infarct extension for other posterior-circulation-dependent brain regions was not independently associated with unfavorable early outcomes. Patients with BAOS had more proximal occlusions and greater infarct volumes in the inferior brain stem. Successful reperfusion (mTICI 2b-3) occurred more often in patients with BAOE than in BAOS (BAOE: 131 (96.3%); BAOS: 47 (83.9%), p = 0.005). Unfavorable early outcomes in patients with BAOS may be explained by a higher likelihood of inferior brain stem infarcts and lower rates of reperfusion success. Basilar artery occlusion due to underlying stenosis is associated with a poorer prognosis than that caused by embolism; these results suggest that aggressive endovascular therapy, usually involving the placement of a permanent stent, may be warranted in these patients. Inferior brain stem and occipital infarcts are prognostically unfavorable in basilar artery occlusion. Basilar artery occlusion due to stenosis occurs more proximally and is associated with worse outcomes. Differentiating etiologies of basilar artery occlusion may influence how aggressively treated the occlusion is.

P029 Successful treatment of Rapidly progressive MDA-5 with dual combination therapy as a bridge to lung transplantation

Abstract Background/Aims Anti-melanoma differentiation-associated gene 5 (MDA5) is known to be associated with rapidly progressive interstitial lung disease (RP-ILD) and is fatal in most cases. The aim of this case report is to highlight the importance of early detection and aggressive treatment in improving the survival of these patients. Methods A 48-year-old Caucasian man, ex-smoker with 20 pack year history with well-controlled asthma, GORD, hiatus hernia and chondrocalcinosis in right knee on hydroxychloroquine was noted to have finger clubbing and decreased air entry at the right lung base on routine follow up. He worked in print finishing for 18 years. Results Investigations: CXR-increased interstitial shadowing. CT chest-extensive fibrosis NSIP pattern with honey combing, traction bronchiectasis throughout both lung fields and ground glass changes at the lower lobes. FVC 2.99 (63% predicted), FEV1 2.82 (73% predicted), FEV1/FVC ratio 0.95%, TLCO 2.75 (25% predicted), KCO 0.70 (46% predicted). CK- 76, Myositis blot- MDA 5 +ve, c ANCA +ve, PR3&amp;MPO -ve and ANA -ve. CRP 70. Bronchoscopy-normal. He received IV methyl prednisolone 1 gm x 3 followed by prednisolone 60 mg daily and tapering dose along with Cyclophosphamide IVI 1 gm x 6 cycles. Long term home oxygen was prescribed. As he did not improve on initial immunosuppressive treatment, Rituximab IVI 1 gm x 2 was administered within two months of initial therapy. He was referred for lung transplant. As maintenance, he was on mycophenolate 1.5 gm BD, prednisolone 10 mg OD, Co-trimoxazole 960 mg x 3 days per week and rituximab 1 gm at six months and 12 months. He had bilateral lung transplant in Cambridge after two years from the diagnosis. Unfortunately, he developed organizing pneumonia, primary graft dysfunction requiring a brief ITU admission and tracheostomy soon after the transplant. His current medications include Prednisolone 5mg, Mycophenolate 2g Tacrolimus 3mg mane, 2mg nocte, Azithromycin 250mg three times weekly and Septrin 480mg od. He is now managing well, cycling 10-15 miles daily. He no longer requires ambulatory oxygen. His most recent FEV1 4.1L, FVC4.9L and resting saturation 97%. Dual combination aggressive immunosuppressive therapy led to significant disease control in this patient, enabling him to be enlisted for and finally to receive a double lung transplant within a reasonable time limit. It has been reported that patients with MDA 5 ILD have a 1-year survival of 67.5% and a 3-year survival of 46.7%. Conclusion Key Learning Points: Patients with MDA5-ILD have significantly high mortality rate but early diagnosis and optimal treatment combination can lead to favourable outcomes. These patients should be monitored for opportunistic infections. Patient commitment is important. Our patient stopped smoking, reduced weight to improve fitness to optimise the transplant success. Disclosure K. Yein: None. S. Sadiq: None. S. Zeb: None. A. Ahmed: None. E. Price: None.

209 Aggressive immunosuppressive therapy is beneficial early on in reducing autoreactive T cell proliferation in Omenn syndrome/Severe combined immunodeficiency

209 Aggressive immunosuppressive therapy is beneficial early on in reducing autoreactive T cell proliferation in Omenn syndrome/Severe combined immunodeficiency

Systemic lupus erythematosus with juvenile onset: current status of the problem (literature review)

Patients with juvenile-onset systemic lupus erythematosus (jSLE) account for up to 25% of all SLE patients. The main difference between jSLE and SLE in adults is the greater role of genetic factors in the pathogenesis, higher activity, earlier development of complications and the need for more aggressive immunosuppressive therapy, which allows us to consider the onset of the disease in childhood as a special phenotype of SLE. The relevance of the study of jSLE arises from the variability of clinical manifestations and the unpredictability of the course, the difficulty of early diagnosis, the rapid development of organ damage and the unfavorable life prognosis.The article presents the most important modern data on the diagnosis, classification, features of the clinical picture and treatment approaches of jSLE from a practical point of view.

Endoscopic closure techniques of bariatric surgery complications: a meta-analysis.

Leaks following bariatric surgery, while rare, are potentially fatal due to risk of peritonitis and sepsis. Anastomotic leaks and gastro-gastric fistulae following Roux-En-Y gastric bypass (RYGB) as well as staple line leaks after sleeve gastrectomy have historically been treated multimodally with surgical drainage, aggressive antibiotic therapy, and more recently, endoscopically. Endoscopic clipping using over-the-scope clips and endoscopic suturing are two of the most common approaches used to achieve full thickness closure. A systematic literature search was performed in PubMed to identify articles on the use of endoscopic clipping or suturing for the treatment of leaks and fistulae following bariatric surgery. Studies focusing on stents, and those that incorporated multiple closure techniques simultaneously, were excluded. Literature review and meta-analysis were performed with the PRISMA guidelines. Five studies with 61 patients that underwent over-the-scope clip (OTSC) closure were included. The pooled proportion of successful closure across the studies was 81.1% (95% CI 67.3 to 91.7). The successful closure rates were homogeneous (I2 = 39%, p = 0.15). Three studies with 92 patients that underwent endoscopic suturing were included. The weighted pooled proportion of successful closure across the studies was shown to be 22.4% (95% CI 14.6 to 31.3). The successful closure rates were homogeneous (I2 = 0%, p = 0.44). Three of the studies, totaling 34 patients, examining OTSC deployment reported data for reintervention rate. The weighted pooled proportion of reintervention across the studies was 35.0% (95% CI 11.7 to 64.7). We noticed statistically significant heterogeneity (I2 = 68%, p = 0.04). One study, with 20 patients examining endoscopic suturing, reported rate of repeat intervention 60%. Observational reports show that patients managed with OTSC were more likely to experience healing of their defect than those managed with endoscopic suturing. Larger controlled studies comparing different closure devices for bariatric leaks should be carried out to better understand the ideal endoscopic approach to these complications.

The Costs of Caring: Quantifying Compassion Fatigue and Compassion Satisfaction in Burn Therapists.

Recovery from a severe burn injury requires early and aggressive therapy, which is often painful and distressing to the patient. Burn therapists who guide these interventions may be prone to experiencing secondary trauma and compassion fatigue through repeated exposure to difficult situations. At the same time, therapists may gain a sense of purpose and altruism from their work, fostering compassion satisfaction. Despite being well documented in other health professions, compassion fatigue and compassion satisfaction have not been sufficiently explored among burn therapists. This study aimed to quantify and characterize burn therapists' compassion fatigue and satisfaction using the Professional Quality of Life scale and to answer the question of what job-related factors may impact these outcomes. A total of 143 burn therapists participated in this study. Results revealed compassion fatigue subsale scores of burnout and secondary traumatic stress in the low or moderate range and compassion satisfaction scores in the moderate or high ranges for all participants. This study presents new knowledge for the field of burn care in its quantification of professional quality of life in burn therapists. While burn therapists experienced moderate-to-high levels of compassion satisfaction in their work, burnout and stress also approached moderate levels. Years of practice was a significant predictor of compassion. Although the reasons for this are not clear, it may be related to increased competence or confidence in practice and may suggest a need for different support strategies for practitioners at each stage of their burn therapy career.

Regular consumption following early introduction of allergenic foods and aggressive treatment of eczema are necessary for preventing the development of food allergy in children.

Over the past two decades, food allergy prevention strategies have shifted from 'delayed introduction' to 'no delayed introduction' to 'early introduction' of allergenic foods. This article reviews important research in this field published in the early 2020s to support future strategies for food allergy prevention. Recent randomized controlled trials (RCTs), systematic reviews, meta-analyses, and real-world studies have reported that early allergenic food introduction, especially peanut and egg, are effective for preventing food allergies. However, there are also reports that food-induced anaphylaxis admission rates in infants are increasing. Early allergenic food introduction by itself is not sufficient to prevent the development of food allergies. Recent RCTs (SPADE study and COMEET study) have demonstrated that continued regular cow's milk consumption after early introduction is important for preventing the onset of cow's milk allergy. Furthermore, an RCT (PACI study) reported that early and aggressive anti-inflammatory topical therapy for eczema can contribute to the prevention of egg allergy by suppressing percutaneous sensitization. Food allergies may be prevented through a combination of early food introduction, regular consumption, and active eczema treatment. Further research is needed to develop well tolerated, effective, and practical strategies to prevent food allergies.

Immune-Mediated Necrotizing Myopathies: Current Landscape.

Immune-mediated necrotizing myopathy (IMNM), characterized by acute or subacute onset, severe weakness, and elevated creatine kinase levels, poses diagnostic and therapeutic challenges. This article provides a succinct overview of IMNM, including clinical features, diagnostic strategies, and treatment approaches. Recent insights highlight the different clinical presentations and therapeutic options of IMNM stratified by autoantibody positivity and type. Additionally, recent findings call into question the reported link between statin use and IMNM. This review synthesizes current knowledge on IMNM, emphasizing its distinct clinical features and challenging management. The evolving understanding of IMNM underscores the need for a comprehensive diagnostic approach that utilizes a growing range of modalities. Early and aggressive immunomodulatory therapy remains pivotal. Ongoing research aims to refine diagnostic tools and therapeutic interventions for this challenging muscle disorder, underscoring the importance of advancing our understanding to enhance patient outcomes.

Fibrocartilaginous Dysplasia of the Proximal Femur in Two Pediatric Patients, Including a Pathologic Fracture in a Patient With McCune-Albright Syndrome

Fibrocartilaginous dysplasia (FCD) is a variant of fibrous dysplasia that often involves the proximal femur in young adults. It has a similar appearance on imaging as other entities but has stippled calcifications within the lesion. The differential diagnosis often includes benign and malignant tumors such as fibrous dysplasia, chondroblastoma, enchondroma, and chondrosarcoma. Histology is required for diagnosis and treatment is typically surgical due to the potential for pain, pathologic fracture, and deformity. We report the clinical presentation, imaging findings, and management of two pediatric patients with fibrocartilaginous dysplasia of the proximal femur to (1) highlight that recognition that fibrous dysplasia may contain cartilage upon frozen section will avoid overly aggressive therapy, and (2) FCD can occur in the McCune-Albright syndrome.