BACKGROUNDThrombotic thrombocytopenic purpura (TTP) patients have increased risk for allergic transfusion reactions (ATR) due to the number of plasma products they require. This study evaluated the efficacy of solvent detergent treated plasma (S/D treated plasma) to reduce ATRs.STUDY DESIGN AND METHODSAll TTP patients who presented from April 2014 to February 2015 and experienced a moderate–severe ATR to untreated plasma with TPE were switched to S/D treated plasma (Octaplas) for their remaining procedures and included in the study. Patient records were retrospectively reviewed.RESULTSThe overall ATR rate per procedure decreased from 35.0% (95% CI = 15.4%‐59.2%) with untreated plasma to 1.4% ([1/73] 95% CI = 0.0%‐7.4%) with S/D treated plasma. The moderate–severe ATR rate decreased from 20.0% ([4/20] 95% CI = 5.7%‐43.7%) with untreated plasma to 0.0% ([0/73] 95% CI = 0.0%‐4.9%) with S/D treated plasma. The overall ATR rate per plasma unit decreased from 2.6% (95%CI = 1.0%‐5.1%) with untreated plasma to 0.1% (95% CI = 0.0%‐0.4%) with S/D treated plasma. No patients experienced VTE while receiving untreated plasma. Four patients experienced VTE events while receiving S/D treated plasma. All patients who experienced a VTE had additional risk factors for VTE.CONCLUSIONS/D plasma has promise as an effective product to reduce the risk of ATRs in TTP patients. Given the high risk of ATR in TTP patients, consideration of S/D plasma instead of untreated plasma for TPE in these patients may be warranted, especially for patients with a history of moderate to severe ATR. More extensive studies are needed to confirm these findings.
Thyroid storm is a potentially lethal complication of hyperthyroidism with increased thyroid hormones and exaggerated symptoms of thyrotoxicosis. First-line therapy includes methimazole (MMI) or propylthiouracil (PTU) to block production of thyroid hormones as a bridge toward definitive surgical treatment. Untreated thyroid storm has a mortality rate of up to 30%; this is particularly alarming when patients cannot tolerate or fail pharmacotherapy, especially if they cannot undergo thyroidectomy. Therapeutic plasma exchange (TPE) is an ASFA category III indication for thyroid storm, meaning the optimum role of this therapy is not established, and there are a limited number of cases in the literature. Yet TPE can remove T3 and T4 bound to albumin, autoantibodies, catecholamines and cytokines and is likely beneficial for these patients. We report a patient with thyroid storm who could not tolerate PTU, subsequently failed therapy with MMI, and was not appropriate for thyroidectomy. TPE was therefore performed daily for 4 days (1.0 plasma volume with 5% albumin replacement and 2 U of plasma). Over the treatment course, the patient's thyroid hormones normalized and symptoms of thyroid storm largely resolved; his T3 decreased from 2.27 to 0.81 ng/mL (normal 0.8-2.0), T4 decreased from 4.8 to 1.7 ng/mL (0.8-1.8), heart rate normalized, altered mental status improved, and he converted to normal sinus rhythm. He was ultimately discharged in euthyroid state. He experienced no side effects from his TPE procedures. TPE is a safe and effective treatment for thyroid storm when conventional treatments are not successful or appropriate.
Objective Use of high-dose cyclophosphamide without hematopoietic stem cell transplant (HSCT) to treat severe aplastic anemia (SAA) has been controversial due to concern for increased infectious toxicity as compared to anti thymocyte globulin (ATG) and cyclosporine (CSA). As children often tolerate dose-intensive therapy better than adults, we sought to perform a detailed retrospective analysis of both treatment response and toxicity in 28 patients less than 22 years of age treated with 29 courses of high-dose cyclophosphamide as the sole form of immunosuppression. Study Design Children and adolescents with SAA who lacked an HLA-matched sibling donor were treated with cyclophosphamide 50 mg/kg/day for 4 consecutive days then received daily G-CSF until neutrophil recovery, transfusion support, and antimicrobial prophylaxis. Results Overall survival was 85%, with hematologic response of 79% and complete response of 66%. Cumulative incidences of bacterial infection (86%) and fungal infection (62%) were high but deaths due to infection were rare, as were clonal evolution (1/28), clinically relevant PNH (1/28), and relapse (2/28). Conclusion Response rates and survival following high-dose cyclophosphamide in pediatric patients with SAA exceed those seen in adults and compare favorably to ATG/CSA with manageable infectious toxicity.
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