Search

Hemophagocytic lymphohistiocytosis (HLH) is a heterogeneous, life-threatening clinical syndrome. There are scarce data on the quality of care in HLH or data comparing treatment patterns and outcomes between different triggers. We aimed to examine quality-of-care indicators and outcomes in adults with various HLH triggers. In this multi-centre retrospective cohort study of adult HLH in the province of Alberta, Canada (1999-2019), we examined quality indicators including diagnostic testing, time to diagnosis and treatment and trigger identification. We also compared treatment regimens and outcomes across HLH triggers. Logistic regression was used to identify predictors of etoposide use. Overall survival (OS) was estimated using the Kaplan-Meier method. We identified 97 patients; 66 (68%) were male. Triggers included malignancy (36%), infection (35%), autoimmune disease (21%) and idiopathic/others (8%). Specialized tests such as sCD25 (53%) and natural killer degranulation assay (19%) were under-performed, as were testing for infectious triggers. Etoposide was administered in only 33 (34%). Neutropenia, hyperbilirubinemia and hyperferritinemia, but not age, sex and comorbidities, were significant predictors of etoposide use. At median follow-up of 32 months, median OS was 18.8 months. Worse OS was seen in malignancy-associated and idiopathic HLH (log-rank P < 0.001). Our study showed low rates of specialized testing such as sCD25 and a low rate of etoposide use. Development of a standardized provincial protocol has the potential to improve quality of care in adult HLH. [ABSTRACT FROM AUTHOR]

Keywords: retrospective chart review; haemophilia A; immune tolerance induction; inhibitor; recombinant factor VIII Fc fusion protein; rescue therapy EN retrospective chart review haemophilia A immune tolerance induction inhibitor recombinant factor VIII Fc fusion protein rescue therapy 19 25 7 02/04/21 20210101 NES 210101 Dear Editor, Prophylactic factor VIII (FVIII) replacement is the current standard of care for severe haemophilia A but approximately 25%-40% of patients develop inhibitors against exogenous FVIII, rendering FVIII replacement therapy ineffective.1 Eradication of high-titre inhibitors involves immune tolerance induction (ITI): repeated, long-term administration of high-dose FVIII.1 Recombinant FVIII Fc fusion protein (rFVIIIFc [ELOCTATE SP ® sp , Sanofi, Waltham, MA]) is the first extended half-life FVIII approved for haemophilia A.2 Case reports and an initial retrospective chart review suggest that rFVIIIFc ITI may lead to faster tolerization than ITI with standard FVIII concentrates.3,4 This letter reports final clinical outcomes of 29 patients (19 included in the initial analysis) with severe haemophilia A undergoing ITI with rFVIIIFc in a real-world setting.4 We performed a retrospective review of patient charts at 13 sites across the United States and Canada, using previously published methods.4 Briefly, de-identified clinical data were collected from patients with severe haemophilia A and historical high-titre inhibitors, who began first-time or rescue ITI with rFVIIIFc between July 2014 and February 2018 and had >=3 months of exposure to rFVIIIFc ITI. Tolerization was defined as a negative Bethesda titre (<0.6 BU/mL), normal FVIII recovery (>=66% of expected) and rFVIIIFc half-life >=6 hours.5 Altogether, 29 rFVIIIFc ITI patients were identified: 10 first-time (Table 1) and 19 rescue patients (Table 2). [Extracted from the article]