Psychosocial burden of type 1 and 2 hereditary angioedema: a single-center Canadian cohort study.

BACKGROUND: Hereditary angioedema (HAE) is a rare but serious disorder associated with a multifaceted burden of illness including a high prevalence of psychiatric symptoms and impaired health-related quality of life (HRQoL). Despite recent efforts to clarify the psychosocial implications of HAE, important gaps still remain. The aim of this study was to characterize the psychosocial burden associated with HAE types 1 and 2. METHODS: Type 1 or 2 HAE patients (n = 17), aged 19 years or older, completed the Depression, Anxiety, Stress Scale (DASS-21) and the DSM-5 cross cutting measures to identify psychiatric symptomatology, Angioedema Quality of Life Questionnaire (AE-QoL) and the Short-Form 36-Item Health Survey version 2 (SF-36v2) to assess disease-related and generic HRQoL respectively, and the Work Productivity and Activity Impairment Questionnaire (WPAI) to measure impact on work productivity and daily activities. Data analyses were conducted using SPSS statistical software (Version 25.0; IBM, Armonk, NY). Descriptive statistics were used to summarize continuous demographics and clinical characteristics and outcomes of interest while frequency distributions were used for categorical variables. T tests were used to compare SF-36v2 domain scores to Canadian norms and sex differences in scale scores. RESULTS: Depression [DASS-21 score = 6.8 ± 10.2; n = 12 (71%)] anxiety [DASS-21 score = 6.2 ± 8.2; n = 13 (76%)] and stress [DASS-21 score = 10 ± 10.2; n = 13 (76%)] were prevalent. Other psychiatric symptoms warranting inquiry included mania (n = 14, 82.4%), anger (n = 14, 82.4%), sleep disturbances (n = 13, 76.5%), somatic symptoms (n = 11, 64.7%) and impaired personality functioning (n = 9, 52.9%). Mean AE-QoL score was 39 ± 18.2. Mean SF-36v2 domain scores were significantly lower than Canadian normative data for the entire sample (p < 0.05). Impairment in work productivity was minimal; mean activity impairment was 20.6% ± 21.1% [n = 11 (64.7%)]. Female participants reported significantly greater HAE-related stress [DASS; t(15) =  - 2.2, p = 0.04], greater HAE-related fears [AEQoL; t(5.6) =  - 2.7, p = 0.04), and lower SF-36v2 domain scores than male patients. CONCLUSIONS: Study findings offer specific, valuable insight into the psychosocial burden of HAE with the potential to improve clinical management of HAE. Best practices for effective management of HAE should include providing holistic care to address the psychosocial and mental health of HAE patients.

Desmopressin in non-severe haemophilia A: Test-response and clinical outcomes in a single Canadian centre review.

INTRODUCTION: Desmopressin is an effective haemostatic agent for patients with non-severe haemophilia A; however, response may differ between patients of similar severity. Responsiveness is classified based on various cut-off values for plasma levels of FVIII post-desmopressin administration. Patients may be classified differently depending on the values chosen. AIM: To classify desmopressin response in non-severe haemophilia A patients with respect to current test-response definitions. Also, to characterize relationships between test response and clinical outcome of desmopressin use. METHODS: Current desmopressin test-response definitions were obtained from the literature. We adopted peak FVIII level (at 1 hour post-administration) ≥50 IU/dL and <20 IU/dL as complete and no response, respectively, thereby satisfying most reported definitions. Test-responses and clinical outcomes of use between 2007 and 2017 for adult mild/moderate haemophilia A patients were reviewed and correlated. RESULTS: All patients classified as complete responders (n = 31; peak FVIII ≥50 IU/dL) and the majority of partial responders (n = 11; peak FVIII ≥20 to <50 IU/dL) had good clinical outcomes after desmopressin use for a variety of bleeding episodes and procedures. Two non-responders (peak FVIII <20 IU/dL) given desmopressin for minor bleeding/procedures also had good clinical outcomes. One patient with a partial test-response (peak FVIII 23 IU/dL) required additional factor concentrate to achieve haemostasis. CONCLUSIONS: Based on our review, we suggest that the determination of desmopressin responsiveness should consider both the change in plasma FVIII levels as well as clinical outcomes associated with prior therapeutic use.

Incidence of catheter-related thrombosis in acute leukemia patients: a comparative, retrospective study of the safety of peripherally inserted vs. centrally inserted central venous catheters.

Central venous catheters are a leading cause of upper-extremity deep vein thrombosis. Concomitant severe thrombocytopenia makes anticoagulation for catheter-related thrombosis (CRT) in patients with acute leukemia (AL) a challenge. Incidence of CRT has been reported to be increased in those with peripherally inserted central catheters (PICC) vs. those with centrally inserted ones (CICC). Our objective is to compare the incidence rate of CRT in leukemia inpatients who received either a PICC vs. CICC. We retrospectively reviewed adult inpatients admitted to hematology wards with a new diagnosis of AL and who received either a PICC or a CICC. Baseline patient and catheter characteristics were recorded. Our primary outcome was the incidence rate of CRT in each group. The secondary outcomes included rates of infectious and mechanical complications. Six hundred sixty-three patients received at least one PICC (338) or CICC (325) insertion. A total of 1331 insertions were recorded, with 82 (11.7 %) and 41 (6.5 %) CRT in the PICC and CICC groups, respectively. The incidence rates were 1.89 and 0.52 per 1000 catheter day in the PICC and CICC groups, respectively. A PICC, when compared to CICC, was a significant risk factor for CRT (sHR 2.5, p < 0.0001). The prevalence and incidence rates of CRT in our AL patients were higher than predicted for a general cancer patient population. These rates were higher in the PICC group compared to the CICC group. We recommend careful consideration of thrombotic and bleeding risks of AL inpatients when choosing a central venous catheter.