Informations générales (source: ClinicalTrials.gov)
Efficacy of Methylprednisolone Pulses in Neuroendocrine Celles Hyperplasia of Infancy : An Early Phase Study
Interventional
Phase 2
Assistance Publique - Hôpitaux de Paris (Voir sur ClinicalTrials)
septembre 2024
avril 2028
11 septembre 2025
Childhood interstitial lung diseases (chILD) are a heterogeneous group of rare and severe
disorders with an estimated prevalence of 1/100,000. Among them, neuroendocrine cells
hyperplasia of infancy (NEHI), also called persistent tachypnoea of infancy (PTI), is one
of the most common aetiology (up to 16% of the cases). NEHI involves young infants
(median age at onset 3 to 6 months) with tachypnoea, hypoxemia, crackles, retractions,
failure to thrive and specific localizations of ground glass opacities (GGO) on chest
CT-scan (paramediastinal areas and anterior lobes (right middle lobe and lingula). At
diagnosis, most patients (50 to 100%) require oxygen supplementation that usually lasts
for months to years, sometimes associated with nutritional support with eventual enteral
nutrition. NEHI is believed to be related to an increased number of neuroendocrine cells
in airway epithelial area. These cells are abundant in foetal life, when they play a role
in regulating the lung development and decrease before birth.
There is no specific treatment for NEHI. The main treatment of chILD is corticosteroids.
However, in NEHI, their efficacy is matter of debate. There is only a few NEHI cases
series or cohorts all over the world, accounting for a maximum of 500 reported cases
within only retrospective studies. Among them, United States and Argentina teams report
supportive care only (oxygen therapy and nutritional support) whereas other teams, like
the French ones largely uses IV corticosteroid pulses.
Unlike the majority of chILD, NEHI prognosis is usually good. However, at school-age, 26%
of the patients remain symptomatic or have an abnormal lung function. Moreover, oxygen
therapy significantly affects quality of life (QoL) of the children with ILD (-10.43/100
points, p=0.02) but also QoL and mood of their parents (unpublished data).
The present study hypothesis that corticosteroids are associated with a reduction of the
length of oxygen support in infants with NEHI.
Etablissements
| Les établissements d'Île-de-France ayant mis à jour leurs données Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| CHI DE CRETEIL | NATHAN Nadia | 18/10/2025 09:58:05 | Contacter | ||
| AP-HP Assistance publique - Hôpitaux de Paris | 18/10/2025 09:58:06 | Contacter | |||
| AP-HP - Hôpital Armand Trousseau-La Roche Guyon | |||||
| AP-HP - Hôpital Necker-Enfants Malades | |||||
| AP-HP - Hôpital Robert Debré | |||||
| Les établissements sans correspondance certaine dans le répertoire FINESS dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| Pediatric Pulmonology Department and Reference centre for rare lung diseases - 75012 - Paris - France | Nadia NATHAN | Contact (sur clinicalTrials) | |||
Critères
Tous
Inclusion Criteria:
- Infant aged under 12 months
- NEHI diagnosis based on:
- The recently validated clinical Liptzin score ≥7/10 associated with a
suggestive thoracic CT pattern with ground glass opacities confined to middle
lobe, lingula, and paramediastinal lung areas OR
- a clinical and thoracic CT suspicion and a lung biopsy showing an increased
number of neuroendocrine cells in the epithelial airways area (at least one
bronchiole with at least 10% of neuroendocrine cells)
- Oxygen requirement (awake and/or asleep) based on the usual pediatric
recommendations (see section 4.1.1)
- Followed in one of the RespiRare participating centers
- Written informed consent of the holder(s) of its legal representative at the
inclusion
- Infant aged under 12 months
- NEHI diagnosis based on:
- The recently validated clinical Liptzin score ≥7/10 associated with a
suggestive thoracic CT pattern with ground glass opacities confined to middle
lobe, lingula, and paramediastinal lung areas OR
- a clinical and thoracic CT suspicion and a lung biopsy showing an increased
number of neuroendocrine cells in the epithelial airways area (at least one
bronchiole with at least 10% of neuroendocrine cells)
- Oxygen requirement (awake and/or asleep) based on the usual pediatric
recommendations (see section 4.1.1)
- Followed in one of the RespiRare participating centers
- Written informed consent of the holder(s) of its legal representative at the
inclusion
- Other cause of chILD assessed by lab biology tests, genetic analysis for surfactant
genes (if available), bronchoalveolar lavage, and/or lung biopsy.
- Patient treated with IV methylprednisolone pulses before (any time)
- Diabetes
- Uncontrolled arterial hypertension
- Absence of Health care insurance
- Ongoing infection
- Immunization with a live attenuated vaccine within the past two weeks
- Long term treatment with Azithromycin and/or Hydroxychloroquine
- Patients already included in an interventional study (RIPH1, clinical investigation
or clinical trial)