Occult Kidney Dysfunction in Children With Transfusion-Dependent Thalassemia

Background: Thalassemia is the commonest hemoglobinopathy in Southeast Asia. Kidney dysfunction is an underreported sequelae in children with thalassemia. We conducted a retrospective study to identify the prevalence of and predisposing factors for kidney dysfunction in children with transfusion-dependent thalassemia (TDT).Method: Abnormal kidney function was defined as children with a glomerular filtration rate (GFR) of <90 ml/min/1.73 m2 or a decline in GFR of >20 ml/min/1.73 m2 or presence of nephrotic range proteinuria within 3 years of commencing regular (every ≤6 weeks) red cell transfusion. Data analyzed were age at diagnosis of thalassemia, number of transfusion-years, iron chelation therapy, serum ferritin, and pre-transfusion hemoglobin levels.Results: Eighty-one children were studied. Mean age was 11.72 ± 5.275 years. Thirty out of 81 (37%) demonstrated abnormal kidney function. Evidence of glomerular hyperfiltration was seen in 29/81 patients (25.85%) at their last clinic visit. This fraction was doubled [48/81 (59.3%)] when the cohort was tracked back by 3 years from the last clinic encounter. Age at diagnosis (RR, 1.157; 95% CI, 1.014–1.319; p = 0.03) and duration of receiving transfusions (RR, 0.984; 95% CI, 0.974–0.994; p = 0.001) were associated with increased risk of developing abnormal kidney function.Conclusion: Abnormal kidney function in children with TDT may be overlooked by medical personnel without active screening measures. Children receiving regular red cell transfusions require systematic surveillance to enable early detection of kidney dysfunction and timely implementation of appropriate therapeutic interventions.

A Case Report of Femoral Hematoma following Obturator Nerve Block

Background. When an obturator nerve block (ONB) is performed, the conventional landmark method or ultrasound-guided method is used. The major complications of this block are hematoma, but there are very few reports of its complications. We encountered massive bleeding and a huge hematoma after ONB. Case Presentation. A 95-year-old female underwent transurethral resection of the bladder tumor. Induction of anesthesia was accomplished via spinal anesthesia and right ONB using the landmark method. Postoperatively, subcutaneous bleeding was detected in the lower right interior thigh. Concentrated red cell transfusion was conducted to address the anemia. There was no subsequent expansion of the hematoma. It resolved on postoperative day (POD) 53. The hematoma was deemed to be inadvertently introduced due to an obturator artery puncture during the obturator nerve block. Conclusions. Close attention is necessary to avoid advancing the needle too deep into the obturator during obturator nerve block.

Predictors of Mortality and Outcomes of Liver Transplant in Spur Cell Hemolytic Anemia

BACKGROUND : Spur cell hemolytic anemia (SCHA) is a rare, acquired, non-immune hemolytic anemia of decompensated cirrhosis resulting from abnormal lipid composition of the red cell membrane. Treatment is limited to red cell transfusion. Data describing prognostic impact, outcomes of liver transplant, and clinical hematologic characteristics of SCHA are absent or limited; published data on SCHA are limited to single-patient case reports and small single-center case series. The independent prognostic impact of SCHA on patients with cirrhosis remains unclear, and it is not known if hemolytic anemia severity has a significant impact on survival of these patients. As a result, SCHA is not formally considered during liver transplant evaluation, and patients do not receive MELD exception points for this diagnosis. METHODS : We performed a multicenter, 24-year observational cohort study of patients with SCHA, retrospectively analyzing hepatic and hematologic parameters, independent predictors of mortality, and long-term outcomes of liver transplant. Strict diagnostic criteria for SCHA were applied, requiring all of the following: (1) decompensated cirrhosis; (2) anemia; (3) objective laboratory evidence of hemolysis; (4) no alternate contributing cause of hemolysis or acanthocytosis; and (5) chart documentation of a confirmed diagnosis of SCHA incorporating peripheral blood film examination. The primary outcome was mortality at 3 months after date of SCHA diagnosis. The impact of hemolytic parameters on 3-month mortality was evaluated utilizing multivariable logistic models. Observed mortality vs. expected mortality (per MELD-Na score and Child-Turcotte-Pugh class) was compared using standardized mortality ratios. Given the limited survival of this population, red cell transfusion dependence was defined as 4 or more units of red cells transfused during the 60-day peri-diagnostic period. RESULTS : Patients: 69 patients with SCHA were included (FIGURE 1). The median (interquartile range) age was 53 (42-59) years; 46.4% were female, and 11 (15.9%) received liver transplant. Alcohol contributed to the etiology of cirrhosis in 53 patients (76.8%). The median (IQR) survival from SCHA diagnosis of patients not receiving liver transplant was 58 (23-113) days. 39 patients (56.5%) were red cell transfusion-dependent. Hematologic parameters are described in TABLE 1. Outcomes of Liver Transplant: All 11 patients undergoing transplant had rapid and complete resolution of SCHA, with an improvement in median hematocrit from 22.1% to 34.6% post-transplant (P=0.001) (TABLE 2) and excellent post-transplant outcomes, with 9 patients still alive after 6.1 years median follow-up. Independent Predictors of Mortality in SCHA: In multivariable logistic models adjusting for age, sex, etiology of cirrhosis, active/recent variceal bleeding, and Child-Turcotte-Pugh score, transfusion dependence had an OR for 90-day mortality of 9.14 (95% CI, 2.46-34.00) and reduced pre-transfusion hematocrit had an OR of 4.73 (95% CI, 1.42-15.82) per 6% decrease; increased red cell transfusion requirement, reduced hemoglobin, increased lactate dehydrogenase, and increased indirect bilirubin were also independently predictive of higher 90-day mortality (FIGURE 2). Performance of MELD-Na and Child-Turcotte-Pugh Scores in Estimating 90-Day Mortality: MELD-Na and Child-Turcotte-Pugh scores consistently significantly underestimated 90-day mortality, with standardized mortality ratios (SMRs) >1 across all scores/classes [MELD-Na 20-29, SMR 2.42 (1.18-4.44); Child-Turcotte-Pugh class B, SMR 4.46 (1.64-9.90)], FIGURE 3. CONCLUSIONS : In this largest study of SCHA to date, SCHA was associated with substantial excess mortality than was predicted by MELD-Na or Child-Turcotte-Pugh scores. Several clinical and laboratory parameters of hemolytic anemia severity, including transfusion burden, hemoglobin, and markers of hemolysis were each independent predictors of 90-day mortality in SCHA. Despite its unique morbidity as a complication of decompensated cirrhosis, outcomes of liver transplant were excellent in all 11 patients undergoing this intervention, with total resolution of SCHA and no evidence of recurrence. These findings should promote greater awareness of SCHA as a clinical entity and broader consideration of MELD exception points for afflicted patients when transplant-eligible. Figure 1 Figure 1. Disclosures Al-Samkari: Novartis: Consultancy; Amgen: Research Funding; Argenx: Consultancy; Rigel: Consultancy; Dova/Sobi: Consultancy, Research Funding; Agios: Consultancy, Research Funding; Moderna: Consultancy.

Hemolytic Uremic Syndrome Caused by Mycoplasma Pneumoniae Infection in Children: A Case Report and Literature Review

We describe the case of a 6-year-old boy with a Mycoplasma pneumoniae (M. pneumoniae) respiratory tract infection associated with thrombotic microangiopathic hemolytic anemia and thrombocytopenia with renal failure which was diagnosed as atypical hemolytic uremic syndrome. Renal biopsy showed features of thrombotic microangiopathy. The patient was treated with azithromycin for the M. pneumoniae infection, and supportive care with red cell transfusion and renal dialysis in the acute period. The microangiopathic hemolytic anemia and thrombocytopenia resolved within 2 months after diagnosis but the renal function damage was irreversible. The patient developed end-stage renal disease and required long term renal replacement therapy.

Systemic Bevacizumab for Refractory Bleeding and Transfusion-Dependent Anemia in Heyde Syndrome

Heyde syndrome, the co-occurrence of aortic stenosis and bleeding gastrointestinal angiodysplasia, is managed with aortic valve replacement. However, severe bleeding and anemia can preclude safe use of the antiplatelet or anticoagulant therapy required for this intervention. We present a case of the novel and successful treatment of severe, refractory bleeding and transfusion-dependence with antiangiogenic therapy in a patient with Heyde syndrome. Following initiation of systemic bevacizumab, the patient achieved durable hemostasis with normalization of hemoglobin, liberation from red cell transfusion and iron infusion dependence, and successful initiation of aspirin therapy where it had previously failed. This facilitated her subsequent successful transcatheter aortic valve replacement. Plasma vascular endothelial growth factor levels, which were monitored during therapy, rose paradoxically after initiation of bevacizumab and normalized after its discontinuation. Given the angiogenic dysregulation of Heyde syndrome, systemic bevacizumab may be an effective and safe targeted therapy for management of refractory gastrointestinal bleeding, thereby facilitating antiplatelet therapy and aortic valve replacement in these challenging cases. Additional investigation into the therapeutic role of angiogenesis inhibition as a hemostatic modality in Heyde syndrome is warranted.

Red cell transfusion benefits in oncology, haematology and palliative medicine populations: a narrative review

Due to the heterogenous nature of the palliative medicine patient population, assessment of benefit, and thus choice of appropriate patient for consideration of transfusion, can be challenging. This can be confounded by the use of both liberal and restrictive transfusion thresholds. The multifactorial nature of many symptoms of anaemia, particularly in patients with advanced malignancy, can further complicate. As such, there is a paucity of data supporting the subjective, objective and clinical benefit of red cell transfusion in the palliative medicine setting. This narrative review summarises the research and evidence surrounding the benefits of red cell transfusion, with a particular emphasis on the oncological, haematological and palliative medicine population. There is a lack of a validated, reproducible patient-reported outcome measures (PROM) to assess response to red cell transfusions in the palliative medicine population with outcome measures varying from objective improvement in haemoglobin level post-transfusion, to subjective response in primary symptom(s). Further investigation is required regarding the development of effective PROMs assessing response to red cell transfusion in the palliative medicine population, to ensure judicious use of this scarce and valuable resource.