Hemangiol, Post Marketing Surveillance Study (postHemangiol)

Prescription of Hemangiol in the Treatment of Proliferative Infantile Hemangiomas Requiring Systemic Treatment: Post Marketing Surveillance Study

Infantile hemangioma is a benign tumor belonging to the group of vascular tumors in the ISSVA classification (International Society for the Study of Vascular Anomalies). The diagnosis is clinical and radiological. The hemangioma appears during the first weeks of life (70% classically within 2 weeks after birth) but can, when it develops in the subcutaneous tissue, appear until the age of 2 to 3 months .

Its evolution is characteristic and is divided into 3 phases with a proliferative phase characterized by a rapid increase in the size of the tumor (up to 6 to 12 months), a phase of stabilization (from 12 to 36 months) with a stopping of the growth of the hemangioma and a regression of its size and a phase of involution with the disappearance of the lesion which may give way to residual fibroadipose tissue, cutaneous telangiectases, scars … The usual complications of haemangiomas occur during the proliferative phase. It is necrosis, ulcerations that can be complicated by bleeding or infection and eventually indelible scarring. Other complications related to the site of development of hemangiomas (amblyopia, astigmatism, upper respiratory obstruction, nasal obstruction, sphincter disorders, eating disorders), hemangiomas destroying structures noble (breast hypodévelopment, alopecia). The aesthetic prognosis can be seriously compromised for facial locations.

Historically, when drug therapy was required, patient management was based on systemic corticosteroids (at doses of 3 to 5 mg / kg / day) in first-line therapy and vincristine as a second-line failure of corticosteroid therapy or when life-threatening is at stake.

In 2014, the high French health authority (HAS) gave Marketing Authorization for Hemangiol 3.75 mg / ml oral solution for the management of infantile proliferative hemangioma requiring first-line systemic treatment, evaluating the actual benefit as important.

The selected indication concerns children from 5 weeks to 5 months with:

• Hemangiomas leading to a vital or functional risk,
• Hemangiomas ulcerated painful and / or not responding to simple care,
• Hemangiomas with a risk of permanent scarring or disfigurement. The 2014 HAS Transparency Commission wishes in its report "to have follow-up data of prescriptions allowing to describe on a representative sample of patients, the characteristics of the treated patients, the indication, the doses and the durations of treatment of this specialty ".

The objective of our study is to describe the use of Hemangiol in current practice in our hospital from 2014 to 2018.

Study Overview

• Status: Completed
• Conditions: Infantile Hemangioma

Detailed Description

This study is retrospective, longitudinal, descriptive and observational. Children aged 0 to 1 years hospitalized for introduction of Hemangiol between January 2014 and November 2018 will be included. Patients refuse the use of medical data will be excluded.

The main objective is to describe the population who received Hemangiol. The secondary objectives is to evaluate

• the effectiveness of Hemangiol on proliferative infantile hemangiomas, -the tolerance of Hemangiol in children with proliferative infantile hemangiomas
• the interest of systematic cardiological consultation in children with proliferative infantile hemangiomas receiving Hemangiol

The following criteria will be collected

Demographic description: age, sex, height, weight

Personal history

Description of the hemangioma:

• organ concerned: skin, liver, parotid, mixed.
• location: head, thorax, pelvis, limbs, diffuse ...
• single or multiple
• indication of treatment: aesthetic, functional, ulcerated, cardiac hyperacidity

Rhythm of follow-up: duration of treatment and observance of treatment

Prescribed dose: 1, 2, 3 or 4 mg / kg / day in 2 doses

Evolution of the hemangioma: progression, regression, stagnation. Needed a second medical therapeutic line by specifying the treatment introduced. Necessity of surgical management by specifying the delay between surgery and the introduction of treatment

Description of serious adverse events: hypotension, bradycardia, bronchospasm, hypoglycaemia. Description of other adverse events.

Number of cardio-pediatric consultations, specifying:

• incidence of diagnosed heart disease through systematic cardiac consultation
• effect on ECG data of increasing dosage of Hemangiol: heart rate, PR duration, QRS duration, QT duration

• Study Type: Observational
• Enrollment (Actual): 500

Contacts and Locations

Study Locations

• France
  • Montpellier, France, 34295
    • UH Montpellier

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Children aged 0 to 1 years hospitalized for introduction of Hemangiol for proliferative infantile hemangiomas.

Description

Inclusion criteria:

- Child from 0 to 18 years old hospitalized for introduction of Hemangiol between January 2014 and November 2018

Exclusion criteria:

- Patients refuse the use of medical data will be excluded.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of adverse events in children with proliferative infantile hemangiomas receiving Hemangiol	rate serious adverse events: hypotension, bradycardia, bronchospasm, hypoglycemia; rate of adverse events	6 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of the regression of proliferative infantile hemangiomas in children receiving Hemangiol	rate of regression of the hemangioma; rate of need for a second therapeutic medical line; rate of need for surgical management	6 days
Incidence of diagnosed heart diseases with systematic cardiac consultation events in children with proliferative infantile hemangiomas receiving Hemangiol	rate of diagnosed heart disease	3 days

Collaborators and Investigators

• Sponsor: University Hospital, Montpellier

Publications and helpful links

General Publications

• Socchi F, Bigorre M, Normandin M, Captier G, Bessis D, Mondain M, Blanchet C, Akkari M, Amedro P, Gavotto A. Hemangiol in infantile haemangioma: A paediatric post-marketing surveillance drug study. Br J Clin Pharmacol. 2021 Apr;87(4):1970-1980. doi: 10.1111/bcp.14593. Epub 2020 Nov 16.

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2019
• Primary Completion (Actual): October 20, 2019
• Study Completion (Actual): October 30, 2019

Study Registration Dates

• First Submitted: September 18, 2019
• First Submitted That Met QC Criteria: September 24, 2019
• First Posted (Actual): September 26, 2019

Study Record Updates

• Last Update Posted (Actual): December 2, 2020
• Last Update Submitted That Met QC Criteria: November 30, 2020
• Last Verified: November 1, 2020

More Information

Terms related to this study

• Keywords: Propranolol; Proliferative infantile hemangiomas; Hemangiol
• Additional Relevant MeSH Terms: Skin Diseases; Neoplasms by Histologic Type; Neoplasms; Congenital Abnormalities; Skin Abnormalities; Neoplasms, Vascular Tissue; Hemangioma, Capillary; Port-Wine Stain; Hemangioma
• Other Study ID Numbers: RECHMPL19_0402

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided
• IPD Plan Description: NC

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No