Surgery for chronic arthropathy in people with haemophilia.

BACKGROUND: Chronic arthropathy is a potentially debilitating complication for people with haemophilia - a genetic, X-linked, recessive bleeding disorder, characterised by the absence or deficiency of a clotting factor protein. Staging classifications, such as the Arnold-Hilgartner classification for haemophilic arthropathy of the knee, radiologically reflect the extent of knee joint destruction with underlying chronic synovitis. Management of this highly morbid disease process involves intensive prophylactic measures, and chemical or radioisotope synovectomy in its early stages. However, failure of non-surgical therapy in people with progression of chronic arthropathy often prompts surgical management, including synovectomy, joint debridement, arthrodesis, and arthroplasty, depending on the type of joint and extent of the damage. To date, management of people with mild to moderate chronic arthropathy from haemophilia remains controversial; there is no agreed standard treatment. Thus, the benefits and disadvantages of non-surgical and surgical management of mild to moderate chronic arthropathy in people with haemophilia needs to be systematically reviewed.  OBJECTIVES: To assess the efficacy and safety of surgery for mild to moderate chronic arthropathy in people with haemophilia A or B. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's Coagulopathies Trials Register, CENTRAL, MEDLINE, Embase, CINAHL, and two trial registers to August 2022. We also handsearched relevant journals and conference abstract books. SELECTION CRITERIA: Randomized controlled trials (RCTs) and quasi-RCTs comparing surgery and non-surgical interventions, for any joint with chronic arthropathy, in people with haemophilia, who were at least 12 years old. DATA COLLECTION AND ANALYSIS: The review authors did not identify any trials to include in this review. MAIN RESULTS: The review authors did not identify any trials to include in this review. AUTHORS' CONCLUSIONS: The review authors did not identify any trials to include in this review. Due to a lack of research in this particular area, we plan to update the literature search every two years, and will update review if any new evidence is reported. There is a need for a well-designed RCT that assesses the safety and efficacy of surgical versus non-surgical interventions for chronic arthropathy in people with haemophilia.

Fetal-onset Congenital Dyserythropoietic Anemia Type 1 due to a Novel Mutation With Severe Iron Overload and Severe Cholestatic Liver Disease.

We report a rare case of severe congenital dyserythropoietic anemia type 1 with fetal onset. Our patient presented with fetal hydrops from 19 weeks of gestation, requiring multiple intrauterine transfusions. At birth, she had severe hemolytic anemia with severe jaundice, and was subsequently transfusion dependent. She eventually developed severe iron overload and fulminant liver failure before her demise at 5 months of age. Genetic testing revealed a novel mutation in CDAN1.

Risk Factors for Mortality in Asian Children Admitted to the Paediatric Intensive Care Unit after Haematopoietic Stem Cell Transplantation.

INTRODUCTION: This study aimed to investigate the risk factors associated with mortality in haematopoietic stem cell transplant (HSCT) patients admitted to our paediatric intensive care unit (PICU) over an 8-year period. MATERIALS AND METHODS: A retrospective chart review was conducted of all HSCT patients requiring PICU admission at our centre (a tertiary care university hospital in Singapore) from January 2002 to December 2010. Chief outcome measures were survival at the time of PICU discharge and survival at 6 months after initial PICU admission. RESULTS: Ninety-eight patients underwent HSCT during this period; 18 patients (18%) required 24 PICU admissions post-HSCT. The overall survival to PICU discharge was 62.5%. Of those who survived discharge from the PICU, 33% died within 6 months of discharge. Non-survivors to PICU discharge had a higher incidence of sepsis (89% vs 33%, P = 0.013) and organ failure as compared to survivors (cardiovascular failure 100% vs 20%, P = 0.0003; respiratory failure 89% vs 20%, P = 0.002; and renal failure 44% vs 7%, P = 0.047). Mortality rates were higher in patients requiring mechanical ventilation (70% vs 14%, P = 0.010) and inotropic support (70% vs 14%, P = 0.010). Mortality in all patients with renal failure requiring haemodialysis (n = 4) was 100%. Presence of 3 or more organ failures was associated with 80% mortality (P = 0.003). CONCLUSION: Sepsis, multiple organ failure and the need for mechanical ventilation, inotropes and especially haemodialysis were associated with increased risk of mortality in our cohort of HSCT patients.

Mortality, length of stay, bloodstream and respiratory viral infections in a pediatric intensive care unit.

OBJECTIVES: We investigated whether diagnostic categories and presence of infections were associated with increased mortality or length of stay (LOS) in patients admitted to a pediatric intensive care unit (PICU). METHODS: A retrospective study of all PICU admissions between October 2002 and April 2016 was performed. Oncologic vs nononcologic, trauma/injuries vs nontraumatic, infectious (gram-positive, gram-negative, fungal bloodstream infections, common respiratory viruses) vs noninfectious diagnoses were evaluated for survival and LOS. RESULTS: Pediatric intensive care unit admissions (n = 2211) were associated with a mortality of 5.3%. Backward binary logistic regression showed that nonsurvival was associated with leukemia (odds ratio [OR], 4.81; 95% confidence interval [CI], 2.2-10.10; P < .0005), lymphoma (OR, 21.34; 95% CI, 3.89-117.16; P < .0005), carditis/myocarditis (OR, 7.91; 95% CI, 1.98-31.54; P = .003), encephalitis (OR, 6.93; 95% CI, 3.27-14.67; P < .0005), bloodstream infections with gram-positive organisms (OR, 5.32; 95% CI, 2.67-10.60; P < .0005), gram-negative organisms (OR, 8.23; 95% CI, 4.10-16.53; P < .0005), fungi (OR, 3.93; 95% CI, 1.07-14.42; P = .039), and pneumococcal disease (OR, 3.26; 95% CI, 1.21-8.75; P = .019). Stepwise linear regression revealed that LOS of survivors was associated with bloodstream gram-positive infection (B = 98.2; 95% CI, 75.7-120.7; P < .0005). CONCLUSIONS: Patients with diagnoses of leukemia, lymphoma, cardiomyopathy/myocarditits, encephalitis, and comorbidity of bloodstream infections and pneumococcal disease were significantly at risk of PICU mortality. Length of stay of survivors was associated with bloodstream gram-positive infection. The highest odds for death were among patients with leukemia/lymphoma and bloodstream coinfection. As early diagnosis of these childhood malignancies is desirable but not always possible, adequate and early antimicrobial coverage for gram-positive and gram-negative bacteria might be the only feasible option to reduce PICU mortality in these patients. In Hong Kong, a subtropical Asian city, none of the common respiratory viruses were associated with increased mortality or LOS in PICU.

A Bone Marrow Aspirate and Trephine Simulator.

INTRODUCTION: Bone marrow aspirate and trephine (BMAT) biopsy is a commonly performed procedure in hematology-oncology practice. Although complications are uncommon, they can cause significant morbidity and mortality. Simulation models are an excellent tool to teach novice doctors basic procedural skills before performing the actual procedure on patients to improve patient safety and well-being. METHODS: There are no commercial BMAT simulators, and this technical report describes the rationale, technical specifications, and construction of a low-cost, easily constructed, reusable BMAT simulator that reproduced the tactile properties of tissue layers for use as a teaching tool in our resident BMAT simulation course. Preliminary data of learner responses to the simulator were also collected. RESULTS: From April 2013 to November 2013, 32 internal medicine residents underwent the BMAT simulation course. Eighteen (56%) completed the online survey, 11 residents with previous experience doing BMAT and 7 without experience. Despite the difference in operative experience, both experienced and novice residents all agreed or strongly agreed that the model aided their understanding of the BMAT procedure. All agreed or strongly agreed that this enhanced their knowledge of anatomy and 16 residents (89%) agreed or strongly agreed that this model was a realistic simulator. CONCLUSIONS: We present a novel, low-cost, easily constructed, realistic BMAT simulator for training novice doctors to perform BMAT.

Use of HF20 membrane in critically ill unstable low-body-weight infants on inotropic support.

BACKGROUND: Initiating continuous renal replacement therapy (CRRT) in infants exposes them to the dual hemodynamic challenges of high circuit extracorporeal volumes and potential membrane reactions, in the case of acrylonitrile AN69 membranes. The use of the new Prismaflex HF20 membrane in hemodynamically unstable low-body-weight infants on inotropic support has not been reported. TREATMENT: We describe the use of the HF20 (Gambro Lundia AB, Lund, Sweden) membrane in four low-body-weight infants (2.3 to 5.4 kg) with multi-organ dysfunction syndrome who were critically ill in the Pediatric Intensive Care Unit (PICU), hemodynamically unstable, and on inotropes. We were able to achieve target volume loss in all infants without compromising their hemodynamic status. Mean arterial pressures were maintained between 39 and 57 mmHg. The relatively low circuit volume of the HF20 set (60 ml) obviated the need for blood prime in the majority; however, when blood prime was required, there was no adverse reaction with the polyarylethersulfone (PAES) membrane. Solute clearance in these small infants was efficient with correction of metabolic acidosis and electrolyte abnormalities. Excellent circuit lifespan (56.3 ± 32.3 h) was observed. CONCLUSIONS: CRRT using the HF20 membrane is safe and hemodynamically well tolerated in high-risk, unstable low-body-weight infants with cardiac dysfunction on multiple inotropes.

Use of Activated Recombinant Factor VII in Severe Bleeding - Evidence for Efficacy and Safety in Trauma, Postpartum Hemorrhage, Cardiac Surgery, and Gastrointestinal Bleeding.

BACKGROUND: Uncontrolled bleeding continues to be a major cause of mortality in trauma, cardiac surgery, postpartum hemorrhage and liver failure. The aim of this paper is to assess the evidence supporting the efficacy of activated recombinant factor VII (rFVIIa) administration in these settings. METHODS: Electronic literature search. RESULTS: Numerous retrospective trials have mostly shown a decrease in blood transfusion requirements with no increase in thromboembolic events (TEE), but major limitations in trial design make generalization difficult. In most retrospective reports rFVIIa has been administered as a last-ditch attempt to control bleeding, when acidosis, hypothermia and coagulation factor depletion may not allow optimal rFVIIa effect. Prospective randomized controlled trials have not shown any effect of rFVIIa on mortality or TEE, although some have shown a reduction in RBC requirement. CONCLUSION: Stipulated transfusion protocols in prospective trials have reduced anticipated mortality among controls and make future trials for mortality effect unlikely in view of large sample size requirements. Establishment of these protocols and rapid hemostasis are likely to have greater benefits than administration of a single agent.