UNASSIGNED: In the CALCIPHYX trial, we investigated hexasodium fytate, an inhibitor of vascular calcification, for the treatment of calcific uraemic arteriolopathy (calciphylaxis), a rare condition characterised by painful, non-healing skin lesions.
UNASSIGNED: In this international, phase 3, randomised, double-blind, placebo-controlled trial, adults with an ulcerated calciphylaxis lesion and pain visual analogue scale (VAS) score ≥50/100 were randomised 1:1 to hexasodium fytate 7 mg/kg or placebo intravenously during maintenance haemodialysis. Primary efficacy outcomes were an 8-item modification of the Bates-Jensen Wound Assessment Tool (BWAT-CUA) and Pain VAS in the intention-to-treat population. ClinicalTrials.gov number: NCT04195906.
UNASSIGNED: Overall, 34/37 patients randomised to hexasodium fytate and 26/34 patients randomised to placebo completed the 12-week randomised treatment period. At Week 12, both groups (hexasodium fytate versus placebo) showed similar improvements in BWAT-CUA (mean [standard deviation (SD)], -5.3 [5.2] versus -6.0 [6.2]; least squares mean difference, 0.3 [96% confidence interval (CI): -2.5, 3.0]; p = 0.88) and Pain VAS (mean [SD], -19.5 [26.9] versus -32.2 [38.5]; least squares mean difference, 11.5 [96% CI: -4.8, 27.8]; p = 0.15). One patient randomised to placebo briefly received hexasodium fytate in error. Serious adverse events through Week 12 included: calciphylaxis-related events leading to hospitalisation (2/38 [5%] versus 11/33 [33%]) and death (1/38 [3%] versus 5/33 [15%]). During the subsequent 12 weeks of open-label hexasodium fytate and 4 weeks of follow-up, there were no additional calciphylaxis-related events leading to hospitalisation. Over the course of the entire trial, deaths were 2/38 [5%] for the hexasodium fytate group and 7/33 [21%] for the placebo group.
UNASSIGNED: In patients with calciphylaxis, BWAT-CUA and Pain VAS improved similarly in hexasodium fytate- and placebo-treated patients; over the course of the entire trial, there were fewer deaths and calciphylaxis-related events leading to hospitalisation in the hexasodium fytate group.
UNASSIGNED: Funded by Sanifit, a CSL Vifor company.

Calciphylaxis, also known as calcific uremic arteriolopathy, is a rapidly progressive, rare, and severe condition characterized by vascular calcification and skin necrosis. The pathophysiology involves cutaneous arteriolar calcification followed by subsequent tissue ischemia and infarction, which eventually causes extremely painful skin lesions. The condition is associated with substantial morbidity due to severe pain, non-healing wounds, increased susceptibility to infections, and frequent hospitalizations. Calciphylaxis is a highly fatal condition with one-year mortality rates greater than 50%, most frequently due to sepsis. This report presents a case of a 63-year-old male with end-stage kidney disease (ESKD) who presented with altered mental status and was found to have notable necrotic skin ulcers on the bilateral anterior thighs, a stage IV sacral decubitus ulcer, and necrotic lesions on the scrotum and penis. This case underscores the importance of maintaining a high clinical suspicion for rare conditions like calciphylaxis in patients with multiple risk factors. Diagnosing the disease earlier in its course may improve outcomes and overall prognosis. Unfortunately, in this case, the patient presented too late into the disease course, and ultimately discussions/placement with palliative care were undertaken.

暂无摘要。

Hyperbaric oxygen (HBO2) treatment is used across a range of medical specialties for a variety of applications, particularly where hypoxia and inflammation are important contributors. Because of its hypoxia-relieving and anti-inflammatory effects HBO2 may be useful for new indications not currently approved by the Undersea and Hyperbaric Medical Society. Identifying these new applications for HBO2 is difficult because individual centers may only treat a few cases and not track the outcomes consistently. The web-based International Multicenter Registry for Hyperbaric Oxygen Therapy captures prospective outcome data for patients treated with HBO2 therapy. These data can then be used to identify new potential applications for HBO2, which has relevance for a range of medical specialties.
Although hyperbaric medicine has established indications, new ones continue to emerge. One objective of this registry study was to identify cases where HBO2 has been used for conditions falling outside of current Undersea and Hyperbaric Medical Society-approved indications and present outcome data for them.
This descriptive study used data from a web-based, multicenter, international registry of patients treated with HBO2. Participating centers agree to collect data on all patients treated using standard outcome measures, and individual centers send deidentified data to the central registry. HBO2 treatment programs in the United States, the United Kingdom, and Australia participate. Demographic, outcome, complication, and treatment data, including pre- and posttreatment quality of life questionnaires (EQ-5D-5L) were collected for individuals referred for HBO2 treatment.
Out of 9726 patient entries, 378 (3.89%) individuals were treated for 45 emerging indications. Post-COVID-19 condition (PCC; also known as postacute sequelae of COVID-19; 149/378, 39.4%), ulcerative colitis (47/378, 12.4%), and Crohn disease (40/378, 10.6%) accounted for 62.4% (n=236) of the total cases. Calciphylaxis (20/378, 5.3%), frostbite (18/378, 4.8%), and peripheral vascular disease-related wounds (12/378, 3.2%) accounted for a further 13.2% (n=50). Patients with PCC reported significant improvement on the Neurobehavioral Symptom Inventory (NSI score: pretreatment=30.6; posttreatment=14.4; P<.001). Patients with Crohn disease reported significantly improved quality of life (EQ-5D score: pretreatment=53.8; posttreatment=68.8), and 5 (13%) reported closing a fistula. Patients with ulcerative colitis and complete pre- and post-HBO2 data reported improved quality of life and lower scores on a bowel questionnaire examining frequency, blood, pain, and urgency. A subset of patients with calciphylaxis and arterial ulcers also reported improvement.
HBO2 is being used for a wide range of possible applications across various medical specialties for its hypoxia-relieving and anti-inflammatory effects. Results show statistically significant improvements in patient-reported outcomes for inflammatory bowel disease and PCC. HBO2 is also being used for frostbite, pyoderma gangrenosum, pterygium, hypospadias repair, and facial filler procedures. Other indications show evidence for improvement, and the case series for all indications is growing in the registry.
RR2-10.2196/18857.

Calcinosis cutis is a common ailment of the skin, caused by the accumulation of calcium salts in the subcutaneous regions, though there are rare case reports from rural Maharashtra region. This condition is usually asymptomatic and might manifest as a single growth or several different-sized growths. Its clinical presentation is observed as nodules or plaques without causing injury to underlying tissues. The condition is reported to be a secondary presentation to trauma, malignancies, and connective tissue diseases and has multifactorial underlying etiologies. Recommendation for treatments including both medical and surgical procedures is contingent upon the manifestation and severity of the condition. The final decision can be based on the results obtained from diagnostic modalities like fine needle aspiration and radiological imaging. This is the case of a 35-year-old male with a swelling on his left foot for the past two years without any associated medical history. The patient was managed by surgical incision and had a good recovery.

Calciphylaxis is a disorder causing ischemic skin necrosis, typically associated with end-stage renal disease or those receiving dialysis. Occurrence is rare in those without end-stage renal disease, and treatment options are limited. This case report describes a patient with calciphylaxis without end-stage renal disease or history of dialysis. Treatment with sodium thiosulfate, a first line option, had to be stopped due to metabolic derangements, limiting the healing process. Diagnosis and treatment of this rare disorder are important to prevent further complications that may result.

UNASSIGNED: Calciphylaxis is a rare disorder associated with significant morbidity and mortality. Data registries are an invaluable source of information for rare diseases. We reviewed cases of calciphylaxis recorded in the Australia and New Zealand Dialysis and Transplant Registry (ANZDATA) and evaluated associations and outcomes of this condition.
UNASSIGNED: Data was obtained on all cases of calciphylaxis reported between 2019 and 2022 in Australian and New Zealand patients on kidney replacement therapy (KRT). This cohort was compared to all patients in the registry who received KRT from 2019 to 2022 without an episode of calciphylaxis. Cox proportional hazards regression including a time-varying covariate for calciphylaxis episode was conducted for mortality with models restricted to patients on dialysis only.
UNASSIGNED: From 2019 to 2022, 333 patients had calciphylaxis episodes reported. Overall incidence rate for patients on dialysis was 4.5 (4.1-5.1) episodes per 1000 patient-years on dialysis. Median age was 63 (interquartile range [IQR]: 55-73) years, 54% were female, 66% had diabetes, 59% were obese (body mass index [BMI] ≥ 30 kg/m2) and 77% were receiving hemodialysis (HD) treatment. Compared to patients without calciphylaxis (n = 46,526), patients with calciphylaxis were more likely to be older, female, and have diabetes, greater BMI, coronary artery, and peripheral vascular disease. The median time to calciphylaxis was 3.2 (IQR: 0.9-6.7) years after KRT commencement. Half of the patients with calciphylaxis died by 12 months from diagnosis. Adjusted hazard ratio (HR) of mortality for patients on dialysis with calciphylaxis <1 year and 1 to 4 years after an episode was 5.8 (4.9-6.9) and 1.5 (1.0-2.1), respectively compared to patients on dialysis without calciphylaxis.
UNASSIGNED: Calciphylaxis is a rare but life-threatening condition in people on KRT with the greatest mortality burden within 12 months of diagnosis.

Calciphylaxis is a unique medical condition characterized by calcification of the medial layer of arterioles and soft tissues in a patient\'s skin at the level of the dermis and subcutaneous adipose tissue. The rate of progression of calciphylaxis is rapid, starting with a reduction of blood flow that leads to ischemic changes in the skin that can manifest as painful cutaneous erythematous nodules or plaques and later as skin ulceration. The majority of patients affected by calciphylaxis have predisposing comorbidities such as end-stage renal disease with a long history of hemodialysis and electrolyte abnormalities in calcium, phosphate, and parathyroid hormone levels. This report presents the case of a 72-year-old female patient on hemodialysis who developed calciphylaxis. The methods for early prognosis (the methods of early diagnosis), including clinical presentation, risk factors, imaging techniques, and laboratory investigations, are discussed. The presented case is particularly noteworthy given the onset of calciphylaxis within a mere three months of initiating hemodialysis, a timeline significantly shorter than the typically observed period in most patients. (The case detailed in this report outlines the rapid onset of calciphylaxis in a patient who was receiving hemodialysis for only three months.) This patient with early-onset calciphylaxis highlights the unpredictable nature of calciphylaxis and the need for increased clinical vigilance even in the initial stages of hemodialysis.

Nonosseous abnormalities are often seen on bone scans and can be related to a wide variety of pathology ranging across vascular, infection, and inflammatory etiology. Diffuse soft tissue radiotracer uptake on bone scans is typically attributed to renal or metabolic derangements. Calciphylaxis is the deposition of calcium in small blood vessels, skin, and other organs leading to vascular obstruction and skin necrosis. It is a rare disorder with unknown pathophysiology. Diagnosis of calciphylaxis is challenging and requires an interdisciplinary approach including clinical findings, laboratory results, medical imaging, and skin biopsy. An early diagnosis is important as the disease is associated with high morbidity and mortality. The purpose of this review article is to highlight the role of bone scintigraphy in the evaluation of calciphylaxis and to correlate the findings with other imaging modalities and histopathology.

UNASSIGNED: Small molecule fibroblast growth factor receptor (FGFR) inhibitors, such as pemigatinib, have been developed for the treatment of cholangiocarcinoma (CCA) with rearrangements or fusions in the FGFR2. FGFR inhibitors (FGFRis) have dermatologic side effects such as dry skin or nail bed damage. However, in very rare instances, a life-threatening vascular calcification disease known as calciphylaxis has been linked to these therapies.
UNASSIGNED: We report a patient with metastatic CCA, who developed calciphylaxis following the start of their pemigatinib treatment. Calciphylaxis is associated with skin lesions and affects the dermal microvasculature in addition to the vascular calcification. This case focuses on the management strategy used for this rare adverse event (AE) as well as the pathology and complicated mechanism of calciphylaxis. We highlight the unclear pathophysiology behind this disease by identifying key players in the signaling and molecular pathways in the microenvironment that are needed to trigger this pathology.
UNASSIGNED: Calciphylaxis is normally associated with advanced renal failure in the setting of high phosphate and calcium. However, the patient we present here did not have advanced renal failure or high calcium levels and calcium dysregulation. As FGFRi use becomes more widespread, the more important it becomes to identify and have a treatment strategy for this rare AE.