HAE is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or hives. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1-INH): HAE with normal C1-INH levels and HAE with limited or insufficient C1-INH levels (C1-INH deficiency). This study will concentrate on children with HAE C1-INH deficiency who have received Takhzyro (Lanadelumab) as prophylactic treatment. The main goal of the study is to assess how well lanadelumab works in children with HAE-C1INH deficiency in everyday life. This will be measured by checking how long children who receive lanadelumab will be free of HAE attacks. Other goals are to understand how children with HAE-C1INH deficiency are being treated with lanadelumab, how well the treatment works for them, how safe it is and how often these children need to use healthcare services (like doctor visits, hospital stays, etc.) because of their condition. The study will only look at data already existing in the participants' medical records. No treatment will be given as part of the study.
Study Type
OBSERVATIONAL
Enrollment
40
This is a non-interventional study.
Hospital de Ninos Sor Maria Ludovica
La Plata, Buenos Aires, Argentina
NOT_YET_RECRUITINGCentro de Alergia e Inmunologia Clinica Mar del Plata
Mar del Plata, Buenos Aires, Argentina
NOT_YET_RECRUITINGHospital de Pediatria S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
Ciudad de Buenos Aires, Buenos Aires F.D., Argentina
NOT_YET_RECRUITINGHospital Italiano de Buenos Aires
Buenos Aires, Ciudad Autonoma de BuenosAires, Argentina
Percentage of Participants who are Free of HAE Attacks
Percentage of participants who are free of HAE attacks will be reported. A HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx).
Time frame: Up to 13 months
HAE Attack Rate of Participants
Time frame: Up to 13 months
HAE Attack-free Rate of Participants Relative to Prior Treatment
Time frame: Up to 13 months
Association Between Covariates of Interest and Attack Free Rate
Association between covariates of interest (e.g., age, sex, family history) and attack-free rate will be determined. Associations between attack free rate and covariates of interest will be modelled, using generalized linear models.
Time frame: Up to 13 months
Number of Participants With HAE Attack Based on Attack Severity
Number of participants with HAE attack based on attack severity (mild, moderate, severe, unknown) will be reported.
Time frame: Up to 13 months
Number of Participants With HAE Attack Based on Location
Number of participants with HAE attack based on location (e.g. extremities, abdominal, facial, laryngeal or genital) will be reported.
Time frame: Up to 13 months
Number of Participants with Treatment History Before use of Lanadelumab
Number of participants with treatment history (type of treatment, duration of treatment, reason for start/ discontinuation/ switch) before use of lanadelumab will be reported.
Time frame: Up to 13 months
Average Duration of Lanadelumab Treatment
Average duration of lanadelumab treatment will be reported.
Time frame: Up to 13 months
Number of Participants Characterized Based on Lanadelumab Treatment Patterns
Number of participants based on lanadelumab treatment patterns (e.g. setting of administration, interval of administration changes/dose modifications, reasons for initiation/discontinuation and modifications) will be reported.
Time frame: Up to 13 months
Number of Participants With Treatments Received Following Lanadelumab Discontinuation
Number of participants with treatments (e.g. type of treatment, reason for selection) received following lanadelumab discontinuation will be evaluated.
Time frame: Up to 13 months
Number of Participants With Healthcare Resource Utilization (HRU) Outcomes
HRU outcomes will include number of healthcare professional (HCP) visits, number of emergency department (ED) visits, number of inpatient hospitalizations, number of participants with admission to and days spent in intensive care unit related to HAE-C1INH, and number of school days missed will be reported.
Time frame: Up to 13 months
Duration of Hospital Stay in Days Related to HAE-C1INH Hospitalizations
Length of hospital stay in days related to HAE-C1INH hospitalizations will be reported.
Time frame: Up to 13 months
Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to 13 months
Percentage of Participants With Injection Site Reactions
Percentage of participants with injection site reactions will be reported.
Time frame: Up to 13 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Sanatorio de la Canada-Cordoba
Córdoba, Argentina
RECRUITINGAP-HM- Hopital de La Timone
Marseille, Bouches-du-Rhone, France
RECRUITINGCHU de Grenoble Alpes - Hopital Couple-Enfant
La Tronche, Grenoble, France
RECRUITINGAP-HP - Hopital Armand Trousseau
Paris, France
RECRUITINGUniversitatsklinikum Ulm
Ulm, Baden-Wurttemberg, Germany
NOT_YET_RECRUITINGUniversitatsklinikum Frankfurt am Main
Frankfurt am Main, Hesse, Germany
NOT_YET_RECRUITING...and 14 more locations