Any grade: Hair color changes (76%), anemia (59%), elevated CPK (56%), fatigue (44%), vomiting (20%), hypophosphatemia (35%), maculopapular/acneiform rash (41%/30%), paronychia (24%), epistaxis (20%), decreased growth velocity (13%)Grade ≥3: Elevated CPK (12%), anemia (10%), maculopapular rash (8%), fatigue (4%), increased ALT (4%), decreased growth velocity (5%). The gene discussed is PIK3C2A; the disease is anemia (phenotype).