Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial

Table 2 Inclusion criteria for patients to participate in the study

1	Diagnosis of chronic (≥ 3 weeks duration) diffuse parenchymal lung disease chILD: a) Genetically^a or b) Histologically^b Diagnosis of chronic (≥ 3 weeks duration) idiopathic pulmonary hemorrhage (hemosiderosis)
2	If chILD genetically diagnosed: patients of all ages (including preterm babies and adults age > 30 years) If chILD histological diagnosed or diagnosis of idiopathic pulmonary hemorrhage: mature newborn (age ≥ 37 weeks of gestation age) to adults (age ≤ 30 years)
3	Patients should be clinically stable during baseline (between visits 1 and 2) for inclusion into the study^c
4	START block: no HCQ treatment in the last 12 weeks STOP block: stable HCQ treatment for at least the last 12 weeks
5	Ability of subject or/and legal representatives to understand character and individual consequences of clinical trial
6	Signed and dated informed consent of the subject (if the subject has the ability) and the representatives (of under-age children) must be available before start of any specific trial procedures

^aSurfactant dysfunction disorders including patients with mutations in SFTPC, SFTPB, ABCA3, NKX2–1, further extremely rare entities with specific mutations; for example, in TBX4, NPC2, NPC1, NPB, COPA, LRBA and other genes
^bChronic pneumonitis of infancy (CPI), desquamative interstitial pneumonia (DIP), lipoid pneumonitis/cholesterol pneumonia, non-specific interstitial pneumonia (NSIP), pulmonary alveolar proteinosis after the exclusion of mutations in granulocyte-macrophage colony-stimulating factor-receptor (GMCSF-R)a/b and GMCSF autoantibodies, usual interstitial pneumonia (UIP), follicular bronchitis/bronchiolitis/lymphogenic interstitial pneumonia (LIP), storage disease with primary pulmonary involvement (e.g., Niemann-Pick)
^cTo determine this, attending physicians can use SpO₂ in room air for patients on room air or on O₂ supplement; the absolute difference in SpO₂ is expected not to be ≥ 5% between visits 1 and 2. For patients on respiratory support, the summary key parameters should not change ≥ 20% between visits 1 and 2 and no major changes in other medications between visits 1 and 2

ISSN: 1745-6215