Epistaxis, Hereditary Hemorrhagic Telangiectasia
Conditions
Keywords
Epistaxis, Hereditary Hemorrhagic Telangiectasia, HHT, Sclerotherapy, Epistaxis (nosebleeds) due to Hereditary Hemorrhagic Telangiectasia
Brief summary
The purpose of this study is to test a novel and tolerable office-based treatment method, sclerotherapy with sodium tetradecyl sulfate, for recurrent epistaxis (nosebleeds) related to Hereditary Hemorrhagic Telangiectasia (HHT) disease.
Detailed description
Ninety percent of patients who suffer from Hereditary Hemorrhagic Telangiectasia (HHT) experience epistaxis which can range from mild to recurrent, severe, life threatening episodes. Current methods to treat significant epistaxis have limitations, namely the need for general anesthesia and repeated treatments. The objective of this study is to test a novel and tolerable office-based treatment method, sclerotherapy with sodium tetradecyl sulfate (STS), for recurrent epistaxis related to HHT. Sclerotherapy is the treatment of vascular lesions by injection with an agent which causes thickening of the vessel wall, obstruction of blood flow, clot formation and collapse of the lesion. Sclerotherapy is an established treatment modality for vascular malformations in the skin, GI tract, genitourinary tract and has been used for lesions in various sites in the head and neck. STS is an anion surfactant (detergent) that is commonly used for sclerotherapy. There are case reports in the literature describing sclerotherapy treatments for epistaxis related to HHT using other agents, but these case reports did not lead to prospective studies. We have performed a pilot study to analyze the tolerability and effectiveness of sclerotherapy with STS in a series of patients with recurrent epistaxis related to HHT. In our series, the treatment was found to be well tolerated and effective, based on patient administered questionnaire and review of clinical data. No complications related to the procedure were noted. Further prospective studies would help elucidate the role of sclerotherapy with STS in the treatment algorithm for recurrent epistaxis related to HHT. Our goal is to conduct a prospective, randomized-controlled trial to test the efficacy and tolerability of sclerotherapy using STS in the treatment of recurrent epistaxis due to HHT. A modified crossover design will be utilized with the intervention group receiving sclerotherapy, plus any additional, previously utilized standard treatment methods needed to control epistaxis. The control group will receive their current standard treatment methods, followed by delayed intervention with sclerotherapy. The primary outcomes will be frequency and severity of epistaxis. Secondary endpoints will be hemoglobin level, tolerability of treatment, additional treatment requirements, and quality of life.
Interventions
DRUGSclerotherapy
3% Sodium tetradecyl sulfate (STS) is mixed with air at a ratio of 4 parts air to 1 part STS for injection into the affected vessels in the nose. Topical anesthetic is applied to the nasal mucosa prior to injections. Once the mixture is ready for injection, the needle is placed into the vessel, in a submucosal fashion, penetrating 1-2 mm, and very small quantities of foam are injected The amount of foam injected into each lesion varies between 0.1 cc to 0.25 cc. Individual injection amounts vary between lesions, patients and treatment sessions. No more than a total of 3 ml of solution is used in each session. During each session, several lesions can be treated, but the total amount of STS used does not exceed 3 cc.
OTHERStandard Treatment
Normal standard of care followed by Drug interevention
Sponsors
American Rhinologic Society
University of Minnesota
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* a clinical diagnosis of HHT based on the Curacoa Criteria * age 18 and older * cognitive ability and willingness to sign the study consent form and complete the study forms and questionnaires
Exclusion criteria
* previous sclerotherapy with Sodium Tetradecyl Sulfate
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Severityof Epistaxis | Change from Baseline to 14 weeks | The primary outcome measure will be severity of epistaxis as measured by the Epistaxis Severity Score (ESS). The ESS, a recently developed online questionarrie that calculates the grading system for epistaxis severity. The higher the score the more severe the nose bleeds are Scale consists of several questions with a range of scale from 0-5 The average score is calculated for a final assessment |
Countries
United States
Participant flow
Pre-assignment details
Each group will get the same therapy in a different order Total number of subjects 18 with 2 incompletes. First period is 6 weeks with a 2 week washout period and 2 period is 6 weeks
Participants by arm
| Arm | Count |
|---|---|
| Entire Study Population | 18 |
| Total | 18 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Withdrawal by Subject | 2 | 2 |
Baseline characteristics
| Characteristic | Entire Study Population |
|---|---|
| Age, Continuous | 51.8 years |
| Curacao criteria Family history: | 9 participants |
| Curacao criteria Visceral Lesions | 9 participants |
| Number of Telangiectasia | 7 Telangiectasia |
| Prior Treatment Cautery | 3 participants |
| Prior Treatment Moisturization | 11 participants |
| Prior Treatment Packing | 4 participants |
| Region of Enrollment United States | 18 participants |
| Sex: Female, Male Female | 9 Participants |
| Sex: Female, Male Male | 9 Participants |
| Telangiectasia type Type 1 | 4 participants |
| Telangiectasia type Type 2 | 13 participants |
| Telangiectasia type Type 3 | 1 participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 18 | 0 / 18 |
| serious Total, serious adverse events | 0 / 18 | 0 / 18 |
Outcome results
Primary
Severityof Epistaxis
The primary outcome measure will be severity of epistaxis as measured by the Epistaxis Severity Score (ESS). The ESS, a recently developed online questionarrie that calculates the grading system for epistaxis severity. The higher the score the more severe the nose bleeds are Scale consists of several questions with a range of scale from 0-5 The average score is calculated for a final assessment
Time frame: Change from Baseline to 14 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sclerotherapy Intervention | Severityof Epistaxis | -0.4578 units on a scale | Standard Deviation 2.8943 |
| Standard Treatment | Severityof Epistaxis | -1.4375 units on a scale | Standard Deviation 4.1993 |