imagem top

2024

ANUÁRIO DO HOSPITAL
DONA ESTEFÂNIA

CHULC LOGOlogo HDElogo anuario

OMALIZUMAB AS AN OFF-LABEL PRESCRIPTION FOR PEDIATRIC PATIENTS

Sofia Cosme Ferreira1,2,3; Mariana Gaspar1,2,4; Inês Sangalho1,2, Miriam Araújo1,2; Paula Leiria Pinto1,2,5

1 - Serviço de Imunoalergologia do Centro Hospital Universitário de Lisboa Central, Hospital Dona Estefânia, Lisboa, Portugal.
2 - Centro Clínico Académico de Lisboa, Lisboa, Portugal
3 - Serviço de Imunoalergologia do Hospital Central do Funchal - Dr. Nélio Mendonça, SESARAM, Funchal, Portugal.
4 - Serviço de Pediatria do Hospital Distrital de Santarém, Santarém, Portugal.
5 - Comprehensive Health Research Center (CHRC), NOVA Medical School, Universidade Nova de Lisboa, Lisboa, Portugal

- Comunicação Oral em Congresso Internacional - Peadiatric Allergy and Asthma Meeting (PAAM)

Background: Omalizumab (OMZ) is a recombinant humanized monoclonal anti-IgE antibody. Current indications for treatment in pediatric patients with urticaria include chronic spontaneous urticária  >12 years old. Any other prescription is considered off-label. We aim to evaluate off-label prescriptions of OMZ in pediatric patients with urticaria.
Method: Retrospective analysis including pediatric patients with chronic spontaneous urticaria under 12 years old or/and chronic inducible urticaria, followed in our outpatient allergy clinic since 2020.
Results: We included 5 patients, 60% male gender, median age was 11 years [10;11] and median onset of symptoms was 8 years [7;9]. The diagnosis of chronic spontaneous urticaria was made in 3 patients and chronic inducible urticaria in all patients: 3 cholinergic, 2 cold and 1 symptomatic dermographism. Angioedema was reported in one patient. Full description of demographics, allergic comorbidities, diagnosis and urticaria workup is shown in Table 1. Four patients started OMZ 300mg 4/4 weeks and one on 150mg 4/4 weeks (average treatment duration 10 months [7-20]). All patients were treated before with H1 antihistamine 4x/day and antileukotriene. Short courses of systemic steroids were necessary in 3 patients. One patient tried cyclosporine 100mg, without success. After starting OMZ, all were able to reduce daily treatment and none resorted to systemic steroids. Dose reduction in controlled urticaria was achieved in 2 patients (mean average of treatment 10 months) and 1 patient was able to stop OMZ after 27 months. The scores on Urticaria Activity Score (UAS7), Urticaria Control Test (UCT) and Dermatology Life Quality Index (DLQI) revealed poor control and quality of life before starting treatment. There was an improvement in all scores after OMZ - Table 1.
Conclusion: In our study, treatment with omalizumab has led to clinical improvement and systemic steroids avoidance. This highlights the potential of omalizumab as an effective treatment option in pediatric patients under 12 years old and in chronic inducible urticaria, although off label.

Palavras Chave: omalizumab, urticaria, pediatricoff-label