Long-term Follow-up Of Patients Research Articles

Clinical manifestations and outcomes of children with bone and joint infections

Purpose: This study aimed to determine the clinical, laboratory, imaging, and bacteriological features of bone and joint infections in children and to identify their characteristic features for early diagnosis. 
 Materials and Methods: In this retrospective study patients diagnosed with osteomyelitis and septic arthritis, based on established guidelines, were included. We collected demographic, clinical, and imaging data, along with inflammatory markers and microbiological data, and any invasive procedures performed by orthopedic surgeons. We also reviewed the development of long-term sequelae, the duration of treatments, and the types of antibiotics used for both oral and parenteral therapy.
 Results: The study group consisted of 25 patients with osteomyelitis, 10 with septic arthritis, and 5 with combined infection (osteomyelitis and septic arthritis). Inflammatory marker levels were abnormal in most children. The most commonly identified organism was the Staphylococcal species. Radiological findings compatible with bone and joint infections were detected in all patients whose magnetic resonance imaging results were available. Invasive procedures were performed in 44%, 90%, and 100% of the patients with osteomyelitis, septic arthritis, and combined infection, respectively. 
 Conclusion: Sensitivity increased when all the markers were used together. Magnetic resonance imaging considered the most informative imaging modality for bone and joint infections provided the highest sensitivity in our study. These sensitive indicators can be helpful for the early diagnosis and long-term follow-up of patients with unclear presentations.

Social and Psychological Long-Term Consequences of NMDA Receptor Encephalitis (SAPIENCE) - from patients’ views to patient-centred care

Background and target population: NMDA receptor encephalitis is a rare autoimmune disease with severe neurological and neuropsychiatric symptoms but typically a good functional neurological outcome. Nevertheless, most patients suffer long-term cognitive, psychological, and social impairments that have a significant impact on their well-being and occupational or educational activities-this is of particular concern given the young age of patients (median 21 years). However, because the disease was only recently discovered (2007), these psychosocial effects have not been systematically studied and the resulting impact on patients is not adequately appreciated. The SAPIENCE study is an international scientific project funded by the European Joint Program that aims to characterize the long-term cognitive and psychosocial consequences of this rare disease. The leader of the consortium is Prof. Carsten Finke, Charite Berlin. The Polish team, coordinated by Prof. Donata Kurpas, is responsible for the development of standard protocols, monitoring and training of staff in the study centers where the data are collected, and the corresponding qualitative data analysis.
 Study Design/Methods: Patients will be extensively studied at three European study sites in three consecutive project levels. Stage 1 includes in-depth on-site interviews and focus groups (n=30). Stage 2 develops a semi-structured short-form interview and includes on-site neuropsychological testing (n=150). Stage 3 includes an international online survey of patient-reported outcome measures (PROMs), patient-reported experience measures (PREMs), and disease progression information (n=300). Stages 1-3 will support each other in developing structured interviews, survey questions, and treatment guidelines.
 Results/Outcomes: At Level 1, we will identify themes and narratives that influence patients' quality of life and their experience of coping with the disease. At Level 2, we will identify neuropsychological outcomes in the largest cohort of NMDAR encephalitis patients in the world to date, including executive function, memory, and spatial navigation performance, which have been identified as key areas of impairment. At Level 3, we will examine subjective outcomes and individual predictors of post-acute disease progression.
 Based on Stages 1-3, we will develop international consensus guidelines for post-acute care of NMDARE patients.
 Impact: This project is the first to bridge the gap between standardized assessment and individual patient experience in the long-term follow-up of patients with autoimmune encephalitis. By incorporating the patient perspective into medical decision-making, current and future NMDAR encephalitis patients will benefit from the implementation of the diagnostic and treatment guidelines developed in this project for patient-centered post-acut

Prognostic impact of atrial cardiomyopathy: Long-term follow-up of patients with and without low-voltage areas following atrial fibrillation ablation

BackgroundAtrial cardiomyopathy is known as an underlying pathophysiological factor in the majority of patients with atrial fibrillation (AF). Left atrial low-voltage areas (LVAs) are reported to coincide with fibrosis and likely represent atrial cardiomyopathy. ObjectiveThe purpose of this study was to delineate differences in the long-term prognosis of patients stratified by the size of LVAs. MethodsThis observational study included 1488 consecutive patients undergoing initial ablation for AF. LVAs were defined as regions with a bipolar peak-to-peak voltage <0.50 mV. The total study population was divided into 3 groups stratified by LVA size: patients with no LVAs (n = 1136); those with small (<20 cm2) LVAs (n = 250) LVAs; and those with extensive (≥20 cm2) LVAs (n = 102). Composite endpoints of death, heart failure, and stroke were followed for up to 5 years. ResultsComposite endpoints developed in 105 of 1488 patients (7.1%), and AF recurrence occurred in 410 (27.6%). Composite endpoints developed more frequently in the order of patients with extensive LVAs (19.1%), small LVAs (10.8%), and no LVAs (5.1%) (P for trend <.0001). Multivariable analysis revealed that LVA presence was independently associated with higher incidence of composite endpoints, irrespective of AF recurrence (modified hazard ratio 1.73; 95% confidence interval 1.13–2.64; P = .011) ConclusionLVA presence and its extent both were associated with poor long-term composite endpoints of death, heart failure, and stroke, irrespective of AF recurrence or other confounders. Underlying atrial cardiomyopathy seems to define a poor prognosis after AF ablation.

Long-term efficacy of ocrelizumab in the treatment of patients with multiple sclerosis – case reports

Treatment of patients with multiple sclerosis, despite the availability of many therapies, remains a challenge. The introduction of ocrelizumab, a highly effective therapy, has significantly improved the control of disease activity. The article discusses four different cases of patients treated with ocrelizumab, including one patient with newly diagnosed relapsing-remitting multiple sclerosis, one case of treatment modification after ineffective first-line therapy, and two cases of long-term follow-up of patients with primary progressive multiple sclerosis. The review of case studies is complemented by magnetic resonance images and the results of tests evaluating treatment effectiveness. In all clinical situations discussed, ocrelizumab proved to be the optimal choice. In addition to inhibiting the clinical and radiological progression of the disease in the long term, it was very well tolerated by patients.

Impact of autotransfusion on recurrence of colorectal cancer liver metastasis: Long-term follow-up of patients undergoing curative intent hepatectomy.

Colorectal cancer liver metastasis (CRLM) occurs in upto 50% of cases and drives patient outcomes. Up-front liver resection is the treatment of choice in resectable cases. There is no consensus yet established as to the safety of intraoperative autotransfusion in liver resection for CRLM. Patients undergoing curative-intent hepatectomy for CRLM at a single quaternary-care institution from 1999 to 2016 were included. Demographics, surgical variables, Fong Clinical Risk Score (FCRS), use of intraoperative auto and/or allotransfusion, and survival data were analyzed. Propensity score matching (PSM) was performed accounting for allotransfusion, extent of hepatectomy, FCRS, and systemic treatment regimens. Three-hundred sixteen patients were included. The median follow-up was 10.4 years (7.8-14.1 years). The median recurrence-freesurvival (RFS) and overall survival (OS) in all patients were 1.6 years (interquartile range: 0.63-6.6 years) and 4.4 years (2.1-8.7), respectively. Before PSM, there was a significantly reduced RFS in the autotransfusion group (0.96 vs. 1.73 years, p = 0.20). There was no difference in OS (4.11 vs. 4.44 years, p = 0.118). Patients in groups of FCRS 0-2 and 3-5 both had reduced RFS when autotransfusion was used (p = 0.005). This reduction in RFS was further found when comparing autotransfusion versus no autotransfusion within the FCRS 0-2 group and within the FCRS 3-5 group (p = 0.027). On Cox-regression analysis, autotransfusion (hazard ratio = 1.423, 1.028-2.182, p = 0.015) remained predictive of RFS. After PSM, there were no differences in FCRS (p = 0.601), preoperative hemoglobin (p = 0.880), allotransfusion (p = 0.130), adjuvant chemotherapy (p = 1.000), immunotherapy (p = 0.172), tumor grade (p = 1.000), use of platinum-based chemotherapy (p = 0.548), or type of hepatic resection (p = 0.967). After matching, there was a higher rate of recurrence with autotransfusion (69.0% vs. 47.6%, p = 0.046). There was also a reduced time to recurrence in the autotransfusion group compared with the group without (p = 0.006). There was no difference in OS after PSM (p = 0.262). Autotransfusion may adversely affect recurrence in liver resection for CRLM. Until further studies clarify this risk profile, the use of intraoperative autotransfusion should be critically assessed on a case-by-case basis only when other resuscitation options are not available.

Critical Endpoints In Dynamic Follow-Up Of Anemia Patients With Percutaneous Coronary Intervention For Acute Coronary Syndrome

Introduction — Identifying critical endpoints during long-term follow-up of patients with coronary artery disease (CAD) and anemia after percutaneous coronary intervention (PCI) would allow optimizing management tactics for this group of patients. Objective — To investigate the risks of developing adverse cardiovascular complications in patients with anemia who underwent revascularization for acute coronary syndrome (ACS) and to assess the most significant factors affecting outcomes. Material and Methods — The study included 298 patients with CAD who had undergone PCI at least two years before enrollment. A survey of patients and a retrospective analysis of their medical records were conducted. The outcome was a composite endpoint (death, myocardial infarction, stroke, transient ischemic attack). Kaplan-Meier curves were constructed to assess the effect of anemia on outcomes. To identify predictors of an adverse outcome, a Cox regression model was built. Results — In the group of patients with ACS who underwent PCI, the incidence of anemia was 8.4%. The incidence of the composite endpoint in patients with anemia was 51.6%, which was significantly different from its value in patients without anemia after 400 days of follow-up (logrank test, p&lt;0.008). After day 1,200, both groups exhibited similar dynamics of adverse outcomes. The following factors were statistically significantly linked to the risk of death within 24 months after PCI: left ventricular ejection fraction (p=0.002), anemia (p=0.009), and chronic kidney disease (p=0.02). Conclusion — We demonstrated that anemia influenced the development of adverse outcomes from day 400 to day 1,200 of a follow-up. Some factors, such as left ventricular ejection fraction and the presence of chronic kidney disease, also had a high predictive power for adverse events.

Analysis of post-COVID symptoms and predisposing factors for chronic post-COVID syndrome.

While there is sufficient information about acute COVID-19, which can cause a multisystemic and fatal disease, post-COVID syndrome and risk factors for this condition remain poorly known. We aimed to identify postCOVID symptoms and risk factors for chronic post-COVID syndrome through this study. This prospective cross-sectional study was conducted on 254 out of 384 COVID-19 patients admitted to our COVID-19 polyclinic between February and April 2021. The patients were questioned with a list of 37 symptoms at the fifth and twelfth weeks after disease onset via phone review, and their acute post-COVID (APC) and chronic post-COVID (CPC) symptoms were recorded. Data on risk factors were collected from the hospital's medical records system. Associations between symptom count in the CPC phase and age, sex, hospitalization, RT-PCR result, specific radiological findings, comorbidities, and long-term medications were evaluated. Two hundred twenty-one patients had APC symptoms, and 138 patients had CPC symptoms. While the most common symptom was fatigue at week five, it was hair loss at week 12. Symptoms were observed significantly less in the CPC phase than in the APC phase (Z= -12.301, p= 0.00). Female sex and the presence of specific radiological findings were significantly associated with the occurrence of CPC symptoms (p= 0.03, p= 0.00, respectively). Long-term use of angiotensin-2 receptor blockers (ARBs) was correlated with a low symptom count in the CPC phase (p= 0.00). Female sex and the presence of specific radiological findings were risk factors for developing CPC. Long-term use of ARBs was associated with a low chronic post-COVID symptom burden. A substantial cluster of multisystemic symptoms was observed in both phases, and this condition highlights the requirement for customized outpatient management that includes long-term follow-up and treatment of COVID-19 patients. Identifying the high-risk patients that will develop persistent symptoms can guide this management.

Long-term follow-up of Parkinsonian patients operated on with deep brain electromodulation without intraoperative microrecording

Background: Deep brain electromodulation (DBEM), also known as deep brain stimulation in different intracerebral targets, is the most widely used surgical treatment due to its effects in reducing motor symptoms of Parkinson’s disease. The intracerebral microelectrode recording has been considered for decades as a necessary tool for the success of Parkinson’s surgery. However, some publications give more importance to intracerebral stimulation as a better predictive test. Since 2002, we initiated a technique of brain implant of electrodes without micro recording and based solely on image-guided stereotaxis followed by intraoperative macrostimulation. In this work, we analyze our long-term results, taking into account motor skills and quality of life (QL) before and after surgery, and we also establish the patient’s time of clinical improvement. Methods: This is a descriptive clinical study in which the motor state of the patients was evaluated with the unified Parkinson’s disease scale (UPDRS) and the QL using the Parkinson’s disease QL questionnaire 39 questionnaires before surgery, in the “on” state of the medication; and after surgery, under active stimulation and in the “on” state. Results: Twenty-four patients with ages ranging from 37 to 78 years undergoing surgery DBEM on the subthalamic nucleus were studied. An improvement of 41.4% in motor skills and 41.7% in QL was obtained. Conclusion: When microrecording is not available, the results that can be obtained, based on preoperative imaging and clinical intraoperative findings, are optimal and beneficial for patients.

Long-term follow-up data of patients with Multiple Pituitary Hormone Deficiency

To present the diagnosis processes, clinical findings, and long-term follow-up of patients with MPHD. Between 1999 and 2015, patients diagnosed with MPHD were evaluated. Clinical presentation, anthropometry, imaging studies, and clinical evolution were analyzed. Hormone status was evaluated, including growth hormone (GH), thyroid-stimulating hormone (TSH), adrenocorticotropic hormone (ACTH), follicle-stimulating hormone/luteinizing hormone (FSH/LH), and prolactin (PRL). Data were assessed using the student's t-test and the Mann-Whitney U test. Spearman's correlation was used for correlations. A p-value < 0.05 was considered statistically significant. Forty-five patients were included; 55.6% were male, the mean age at presentation was 5.6 ± 3.9 (0-14.4) years, and the median bone age was 3.5 ± 2.3 (0.5-11) years. At admission, GH deficiency was found in 88.9% of the cases, TSH deficiency in 77.8%, ACTH deficiency in 33.3%, FSH/LH deficiency in 22.2%, and PRL deficiency in 17.8%. During the follow-up, 62% of the cases added other hormone deficiencies. The mean follow-up period was 9.18 ± 3.6 (3.02-17.2) years. Patients with MPHD have very different clinical presentations, with GH and TSH deficiency being the most common in this study. Additional hormonal deficiencies can occur even years after the initial diagnosis and our results demonstrate that genetic height potential is achieved with GH treatment.

Systemic-to-pulmonary artery shunt treated with transcatheter arterial embolization and subsequent lung segmentectomy

BackgroundSystemic-to-pulmonary artery shunt (SPAS) is a rare condition that can occur as a result of congenital heart disease or chronic pulmonary inflammation, occasionally leading to life-threatening hemoptysis. Computed tomography (CT) imaging is crucial in the diagnosis of SPAS, and the optimal management approach for SPAS remains uncertain. This case report presents a novel approach to the treatment of SPAS, consisting of transcatheter arterial embolization of the systemic artery followed by lung segmentectomy.Case presentationA 42-year-old man with abnormal chest findings was referred to us and a diagnosis of SPAS was established based on the CT findings showing a blood flow regurgitation from the dilated left 4th intercostal artery to the Lt. A6. The patient was asymptomatic but we decided to treat him to prevent a risk of future hemoptysis. Transcatheter arterial embolization (TAE) of systemic arteries followed by S6 segmentectomy was successfully performed with minimal blood loss and complete removal of the dilated intra-pulmonary blood vessels. Histological analysis confirmed the diagnosis of SPAS.ConclusionWe reported a case of SPAS, who was successfully treated with the combination of TAE and subsequent segmentectomy. The blood loss during surgery was minimal and this strategy appeared to minimize future recanalization and hemoptysis. Further studies and long-term follow-up of SPAS patients are required to establish standardized management guidelines for this rare condition.

Post-Operative Complications Associated with Long-Term NSAID or Long-Term Opioid Use Prior to Lumbar Spinal Fusion Surgery

ObjectiveLower back pain (LBP) has been implicated as a significant cause of chronic pain in the United States, often requiring analgesic use. In this study, we investigate the trends in long-term preoperative NSAID (LTN) and Opioid (LTO) use in patients with low back pain in the United States, and the resultant postoperative complications following lumbar fusion. MethodsIn this retrospective cohort study of patients with lumbar pathologies, multivariate population-based regression models were developed using the 2010–2017 National Readmission Database. Short-term complications (30-, 90-day) and long-term complications (180-, 300-day) were analyzed at readmission. ResultsOf patients diagnosed with LBP (N = 1427,190) we found a rise in LTO users and a fall in LTN users following 2015. We identified 654,264 individuals who received a lumbar spine fusion, of which 22,975 were LTN users and 11,213 were LTO users. LTO users had significantly higher total inpatient charges (p-value<0.0001) and LOS (p-value<0.0001), while LTN users had lower rates of acute infection (OR: 0.993, 95% CI: 0.987–0.999, p = 0.017) and acute posthemorrhagic anemia (OR: 0.957, 95% CI: 0.935–0.979, p < 0.001) at primary admission. Readmission analysis showed that LTN use had significantly lower odds of readmission compared to LTO use at all time points (p < 0.01 for all). LTN use had significantly higher odds of hardware failure (OR: 1.134, 95% CI: 1.039–1.237, p = 0.005) within 300-days of receiving a lumbar fusion. ConclusionsLTO users had significantly higher readmission rates compared to LTN. In addition, we found that LTN use was associated with significantly higher odds of hardware failure at long-term follow-up in patients receiving lumbar fusion surgery.

Abdominal viscera in the thoracic cavity: a case of large Bochdalek hernia in an adult

Introduction: Bochdaleck hernias (BH) are a congenital defect in the diaphragm's postero lateral part that may cause the abdominal viscera's protrusion into the chest cavity. This type of hernia is rare in adults. Most cases are referred to the emergency department with gastrointestinal obstruction and visceral strangulation symptoms. Case presentation: A 50-year-old man was referred to the emergency department complaining of abdominal pain in the left upper quadrant, intermittent vomiting, and pain in the left hemithorax from one day ago. We found a large diaphragmatic hernia on the left side during evaluation, which causes intra-abdominal organ shift to the left hemithorax. The patient underwent surgery for diaphragmatic herniorrhaphy. Conclusion: Congenital diaphragmatic Bochdaleck hernias are rare and are associated with nonspecific symptoms in adults. They may be symptom-free during the patient's life as in our case. It is necessary to investigate more cases and long-term follow-ups of patients, to improve the diagnosis and treatment of these patients.

Retrospective clinical and genetic analysis of COL6-RD patients with a long-term follow-up at a single French center.

Collagen type VI-related dystrophies (COL6-RD) are rare diseases with a wide phenotypic spectrum ranging from severe Ullrich's congenital muscular dystrophy Ullrich congenital muscular dystrophy to much milder Bethlem myopathy Both dominant and recessive forms of COL6-RD are caused by pathogenic variants in three collagen VI genes (COL6A1, COL6A2 and COL6A3). The prognosis of these diseases is variable and difficult to predict during early disease stages, especially since the genotype-phenotype correlation is not always clear. For this reason, studies with long-term follow-up of patients with genetically confirmed COL6-RD are still needed. In this study, we present phenotypic and genetic data from 25 patients (22 families) diagnosed with COL6-RD and followed at a single French center, in both adult and pediatric neurology departments. We describe three novel pathogenic variants and identify COL6A2:c.1970-9G>A as the most frequent variant in our series (29%). We also observe an accelerated progression of the disease in a subgroup of patients. This large series of rare disease patients provides essential information on phenotypic variability of COL6-RD patients as well as on frequency of pathogenic COL6A gene variants in Southern France, thus contributing to the phenotypic and genetic description of Collagen type VI-related dystrophies.

Diagnosis, combined treatment, and long-term follow-up of a thymoma patient

Thymic neoplasms account for 20 – 25% of primary mediastinal tumors. Nonspecific clinical symptoms are often associated with delayed diagnosis of thymoma, leading to a distinctive problem of late diagnosis. Nowadays, surgical removal of the tumor is the method of choice for thymoma treatment. Combination treatment is indicated in the case of an unresectable tumor. Modern therapy (various combinations of anticancer drugs) helps reduce neoplasm size, stop metastasis, and maintain good quality of life in patients with thymoma.Aim. The presented clinical case of type B3 thymoma (with invasion into surrounding organs and metastatic pleura) is characterized by a long asymptomatic period of the disease, the onset of symptoms after suffering COVID-19 (COronaVIrus Disease 2019) and successful long-term polychemotherapy. The causes of late diagnosis, as well as insufficient oncologic vigilance in evaluating the results of laboratory and instrumental tests, are discussed based on this clinical case.Conclusion. A correct diagnosis, as well as successful treatment, requires a multidisciplinary approach involving physicians from different specialties – general practitioners, oncologists, surgeons, radiologists, and morphologists.

Reduced stress perfusion in myocardial infarction with nonobstructive coronary arteries

Myocardial infarction with nonobstructive coronary arteries (MINOCA) has several possible underlying causes, including coronary microvascular dysfunction (CMD). Early cardiovascular magnetic resonance imaging (CMR) is recommended, however cannot provide a diagnosis in 25% of cases. Quantitative stress CMR perfusion mapping can identify CMD, however it is unknown if CMD is present during long-term follow-up of MINOCA patients. Therefore, this study aimed to evaluate presence of CMD during long-term follow-up in MINOCA patients with an initial normal CMR scan. MINOCA patients from the second Stockholm myocardial infarction with normal coronaries study (SMINC-2), with a normal CMR scan at median 3 days after hospitalization were investigated with comprehensive CMR including stress perfusion mapping a median of 5 years after the index event, together with age- and sex-matched volunteers without symptomatic ischemic heart disease. Cardiovascular risk factors, medication and symptoms of myocardial ischemia measured by the Seattle Angina Questionnaire 7 (SAQ-7), were registered. In total, 15 patients with MINOCA and an initial normal CMR scan (59 ± 7 years old, 60% female), and 15 age- and sex-matched volunteers, underwent CMR. Patients with MINOCA and an initial normal CMR scan had lower global stress perfusion compared to volunteers (2.83 ± 1.8 vs 3.53 ± 0.7 ml/min/g, p = 0.02). There were no differences in other CMR parameters, hemodynamic parameters, or cardiovascular risk factors, except for more frequent use of statins in the MINOCA patient group compared to volunteers. In conclusion, global stress perfusion is lower in MINOCA patients during follow-up, compared to age- and sex-matched volunteers, suggesting that CMD may be a possible pathophysiological mechanism in MINOCA.Clinical Trial Registration: Clinicaltrials.gov identifier NCT02318498. Registered 2014-12-17.

Long-Term Follow-Up of Patients With Elevated Aldosterone-to-Renin Ratio but Negative Confirmatory Test: The Progression of Primary Aldosteronism Phenotypes.

About 10% of patients with arterial hypertension have a positive screening test for primary aldosteronism (PA) and 50% to 70% of them have a negative confirmatory test: the appropriate follow-up of these patients is currently unknown. We investigated the incidence of PA in patients with previous negative confirmatory testing, after at least a 2-year follow-up. One hundred eighty-four patients with a previously elevated aldosterone-to-renin ratio followed by a negative confirmatory test were recruited in 2 hypertension centers (Torino and Munich). We repeated the screening test for PA and, if positive, the confirmatory test (seated saline infusion test or captopril challenge test). Primary end point of the study was the incidence of newly diagnosed overt PA, as defined by a positive confirmatory test. After a mean follow-up of 5 years, 20% of patients developed overt PA. When subtype diagnosis was offered systematically, one-third of patients displayed unilateral PA. Patients who developed PA showed worsening of blood pressure control and a higher rate of cardiac organ damage, despite similar implementation of antihypertensive therapy, compared with patients without PA. A mild progression of autonomous aldosterone secretion was evident even in patients without confirmed PA but with relatively stable control of blood pressure levels over time. About one-fifth of patients with a negative confirmatory test develop overt PA over time. A clinical follow-up of patients with a negative confirmatory test is advisable, along with the repetition of PA investigation, primarily in patients with worsening of blood pressure control.

Long-term follow-up of patients with acute myeloid leukemia undergoing allogeneic hematopoietic stem cell transplantation after primary induction failure

Primary induction failure (PIF) in acute myeloid leukemia (AML) patients is associated with poor outcome, with allogeneic hematopoietic stem cell transplantation (HCT) being the sole curative therapeutic option. Here, we retrospectively evaluated long-term outcomes of 220 AML patients undergoing allogeneic HCT after PIF who never achieved remission, and identified clinical and molecular risk factors associated with treatment response and ultimate prognosis. In this high-risk population, disease-free survival was 25.2% after 5 years and 18.7% after 10 years, while overall survival rates were 29.8% and 21.6% after 5 and 10 years of HCT, respectively. 10-year non-relapse mortality was 32.5%, and 48.8% of patients showed disease relapse within 10 years after allogeneic HCT. Adverse molecular risk features determined at initial diagnosis, poor performance status at the time of allogeneic HCT, and long diagnosis-to-HCT intervals were associated with unfavorable prognosis. Collectively, our data suggests that immediate allogeneic HCT after PIF offers long-term survival and cure in a substantial subset of cases and that high-risk AML patients who never achieved complete response during induction might benefit from early donor search.

Parent-Reported Health-Related Quality of Life Outcomes and Impact on Family of Congenital Diaphragmatic Hernia Survivors After Repair: A Cross-Sectional Comparison to Healthy Controls

BackgroundCongenital diaphragmatic hernia (CDH) survivors often experience long-term CDH-associated morbidities, including musculoskeletal, gastrointestinal and respiratory issues. This study evaluates parent-reported health-related quality of life (HRQOL) and family impact of the disease. MethodsElectronic medical records (EMR) were reviewed and phone surveys performed with parents of CDH survivors who underwent repair at our institution from 2010 to 2019. They completed the following Pediatric Quality of Life Inventory™ (PedsQL™) questionnaires: Generic Core Scales 4.0 (parent-proxy report) and Family Impact (FI) Module 2.0. Age-matched and gender-matched healthy controls from an existing database were used for comparison. Subgroup analysis of CDH patients alone was also performed. Appropriate statistical analysis was used with p < 0.05 significance. Data are reported as median (range). Ethical approval was obtained. ResultsOf 76 CDH survivors, 45 parents (59 %) consented for study participation. Patients and controls were aged 6 (3–12) years; 51 % were male. In Core Scales, there were significant differences between the groups in the overall scores (p = 0.003) and the psychosocial health sub-scores (p = 0.004), but no difference in the physical health domain scores. In the Family Impact Module, there were no significant differences between the groups in overall scores, in parent HRQOL and in family functioning sub-scores. However, CDH patients with learning disabilities had significantly worse Family Impact Module overall scores compared to CDH patients without learning disabilities (p = 0.04). ConclusionThe overall HRQOL in children with CDH survivors is worse compared to controls, but impact on family appears similar. We highlight the need for long-term follow-up of CDH patients, especially those with learning disabilities. Healthcare providers should formulate tailored care plans to address patient and caregiver needs. Type of studyProspective Study. Level of evidenceLevel III.

Treatment of malignant diseases with phytocannabinoids: promising observations in animal models and patients

Amazingly, almost 50 years after the first demonstration of anticancer effects of cannabinoids in vitro and in vivo, well-designed clinical trials that definitively prove tumour-inhibiting effects in man are still missing. Whereas a large number of preclinical studies exist that describe tumour-inhibiting effects of cannabinoids, alone or in combination, but also in the form of medical cannabis or natural extracts in vitro, the number of in vivo studies is still limited. Even more limited are well-documented experiences in man. Most animal studies and experience with cannabinoids in man concern brain tumours. This review summarises the effects of phytocannabinoids in brain, breast, colorectal, head and neck, haematological, liver, lung, pancreatic, ovarian, prostate, and skin cancers in animal models and, if available, in patients. The large majority of animal studies demonstrate tumour-inhibiting effects of cannabinoids, thus confirming in vitro data. Experiences in cancer patients are almost exclusively limited to individual case reports and case series without a control group. Many questions are currently unanswered such as the role of pure cannabinoids compared to combinations, cannabinoids as the eventual sole cancer therapy, optimal dosages, or duration of treatment. Pure cannabidiol (CBD) seems to be superior to pure delta-9-tetrahydrocannabinol (THC) in experimental settings. The role of medical cannabis or extracts is less clear as they vary in their phytochemical composition. In conclusion, cannabis/cannabinoids may slow the progression of tumours. However, the hope that cannabinoids could eventually cure cancer as often spread in social media, is, at present, wishful thinking. Above all, well-designed clinical trials paired with long-term follow-up of cancer patients are needed.

Extended long-term follow-up of metastatic melanoma patients treated with immunotherapy: late relapses and second primary melanomas.

Immunotherapy has revolutionized the treatment of patients with advanced melanoma as well as other cancers. Most studies, whether of interleukin-2 or checkpoint inhibitor therapies, have limited follow-up after 5 years, making the incidence of late relapses uncertain. In addition, the incidence of second primary melanomas in patients with stage IV melanoma treated with immunotherapy has rarely been reported. We performed a single-institution retrospective study of stage IV melanoma patients treated with interleukin-2 or checkpoint inhibitors over the period from 1992 to 2013. We found 59 patients alive and in remission 5 years after the beginning of immunotherapy and reviewed their subsequent clinical course. This 59-patient cohort had a median follow-up of 13.1 years, with 36 patients followed up for at least 10 years. Four patients (6.8%) had relapses of their metastatic melanoma at 5, 8, 15, and 17 years after starting the successful immunotherapy. Three of the four are still alive. Only one patient in 690 patient-years of observation had a second primary invasive melanoma. Although late relapses after immunotherapy for melanoma do occur, we can conclude that the prognosis of stage IV melanoma patients in continuous remission 5 years after starting immunotherapy is excellent, with a progression-free survival of approximately 85% and a melanoma-specific survival of approximately 95% at 20 years in our series. Our incidence of second primary melanomas is lower than usually reported. These results have important implications regarding the follow-up of stage IV melanoma patients successfully treated with immunotherapy.