Patient-Reported Outcomes of Bleomycin Sclerotherapy for Low-Flow Vascular Malformations and Predictors of Improvement

Abstract

Purpose: There is paucity of data on patient-perceived outcomes of bleomycin sclerotherapy for low-flow vascular malformations. In this study, the long-term outcomes of bleomycin sclerotherapy were investigated in terms of quality of life (QoL) and patient-perceived changes in health.

Materials and methods: A cohort of Dutch patients with vascular malformations treated with bleomycin sclerotherapy (June 2010-November 2015) completed a questionnaire evaluating disease symptoms, QoL (Short Form 36), patient-perceived change in health status (Global Rating of Change scales) and treatment satisfaction. QoL was assessed for the patient's status before and after treatment and was analyzed relative to an age and sex-matched Dutch reference population. Predictive factors associated with QoL and patient-perceived improvement in overall health status were assessed using multivariable linear and logistic regression analyses, respectively.

Results: Seventy-seven patients, with a median follow-up of 22 months, were enrolled. About half of the respondents (49.3%) indicated that they perceived (any form of) improvement in their overall health status. Most often improved were the specific health aspects 'pain' (54.5%) and 'overall severity of symptoms' (57.1%). No factors were significantly predictive for patient-perceived improvement in health with respect to the vascular malformation. Impairment in work- or study-related activities prior to sclerotherapy was found to negatively impact physical QoL at follow-up (p = 0.03).

Conclusion: Approximately half of patients with low-flow vascular malformations indicate an improvement in overall health status following bleomycin sclerotherapy, particularly concerning pain and severity of symptoms. However, most patients only perceived little to moderate improvement to their health and desire further treatment.

Keywords: Bleomycin; Lymphatic malformations; Sclerotherapy; Vascular malformations; Venous malformations.

Fig. 1

Flowchart of patient enrollment

Fig. 2

Bar chart of Z-scores of SF-36 domains corrected for age and sex, compared to Dutch reference population mean. The Z-scores or ‘standard mean scores’ indicate how many standard deviations the patient’s score is from the mean of the Dutch reference population. A Z-score of zero (horizontal line) therefore indicates a score conform the reference population. A negative score indicates a lower and a positive score a higher score than the reference population mean.* indicates a significant difference compared with the Dutch reference population. The dotted horizontal lines at standard mean scores of 0.2, 0.5 and 0.8 represent small, moderate and large deviations from the reference population, respectively. Baseline = retrospectively assessed baseline SF-36 score for situation prior to bleomycin sclerotherapy. Follow-up = SF-36 scores after bleomycin sclerotherapy, at the time of completing the questionnaire (median follow-up 22 months). PF = physical functioning, RP = role physical, BP = bodily pain, GH = general health, VT = vitality, SF = social functioning, RE = role emotional, MH = mental health