Neoplasms

At Queen Silvia Children’s Hospital researchers are testing novel anti‑cancer agents and combination regimens to increase cure rates while limiting long‑term side effects in children with solid and hematologic malignancies.

  • Acute Lymphoblastic Leukaemia
  • Acute Myeloid Leukaemia
  • Brain Tumor
  • Medulloblastoma
  • Neuroblastoma
  • Ewing Sarcoma
  • Rhabdomyosarcoma
  • Lymphomas (Hodgkin and non‑Hodgkin)
  • Atypical Teratoid/Rhabdoid Tumour

The site hosts 44 active trials with 68 investigators, many of which are multinational collaborations focused on improving event‑free survival and quality of life.

Hemic and Lymphatic Diseases

Studies here assess blood‑forming disorders and lymphoid cancers, seeking more effective and less toxic treatment options for young patients.

  • Acute Lymphoblastic Leukaemia
  • Acute Myeloid Leukaemia
  • Chronic Graft‑Versus‑Host Disease
  • Anemia associated with Chronic Kidney Disease
  • Lymphoblastic Lymphoma
  • Anaplastic Large Cell Lymphoma (ALCL)

Research aims to boost remission rates and reduce relapse by integrating targeted agents and immunotherapies into standard chemotherapy backbones.

Immune System Diseases

The department investigates disorders driven by immune dysregulation, evaluating both disease‑modifying drugs and supportive therapies.

  • Chronic Graft‑Versus‑Host Disease
  • Proteinuric Glomerular Diseases (FSGS, IgAN, MCD, IgAV, Alport Syndrome)
  • Inflammatory Myofibroblastic Tumor
  • C3 Glomerulopathy

Trials often compare novel complement inhibitors or renin blockers with established ACE inhibitors to protect kidney function.

Digestive System Diseases

Researchers focus on improving nutrition and intestinal health in vulnerable pediatric populations, testing therapies that enhance gut absorption.

  • Intestinal Malabsorption in Preterm Infants
  • Ulcerative Colitis

The goal is to shorten time to full enteral feeding and achieve sustained remission with fewer steroids.

Congenital, Hereditary, and Neonatal Diseases

Efforts target rare genetic conditions, aiming to modify disease progression and improve functional outcomes from early childhood.

  • Congenital Myopathy
  • Dravet Syndrome
  • Mucopolysaccharidosis Type II (Hunter Syndrome)
  • Niemann‑Pick Type C Disease
  • GM1/GM2 Gangliosidoses

Studies include gene‑transfer approaches, enzyme replacement, and small‑molecule therapies designed to cross the blood‑brain barrier.

Musculoskeletal Diseases

Clinical work seeks to restore muscle strength and mobility in children affected by inherited muscular disorders and tumor‑related muscle disease.

  • Duchenne Muscular Dystrophy
  • Congenital Myopathy
  • Rhabdomyosarcoma

Trials evaluate exon‑skipping agents, micro‑dystrophin gene therapy, and novel chemoradiation schedules to preserve limb function.

Nervous System Diseases

The center conducts research on brain and spinal cord disorders, testing interventions that protect cognition and reduce tumor‑related damage.

  • Brain Tumor
  • Medulloblastoma
  • Dravet Syndrome
  • Atypical Teratoid/Rhabdoid Tumour

Projects include lithium neuroprotection after radiotherapy and precision‑medicine trials using targeted inhibitors for rare CNS tumors.

Pathological Conditions, Signs and Symptoms

Investigations address acute clinical complications and symptom‑focused outcomes, striving to improve recovery speed and quality of life.

  • Acute Kidney Injury after Cardiac Surgery
  • Facial Nerve Palsy (Bell’s Palsy)
  • Proteinuric Glomerular Diseases

Therapeutic strategies such as peptide hormones and anti‑inflammatory agents are being compared with standard care to reduce organ injury.

Behavioral and Psychological Research

Studies explore neuropsychiatric syndromes and immune response after cancer treatment, aiming to restore mental health and vaccine efficacy.

  • Pediatric Acute‑Onset Neuropsychiatric Syndrome (PANS)
  • Immunity after Revaccination (Varicella, Measles)
  • Cognitive Processing Speed after Brain Radiotherapy

Outcomes focus on symptom severity scales, antibody titers, and neurocognitive testing to guide supportive interventions.

Therapeutic Techniques and Equipment

The site evaluates cutting‑edge diagnostic tools and delivery systems to enhance precision of pediatric therapies.

  • Anti‑angiogenic Treatment Response Monitoring
  • Advanced Imaging for Tumor Volume Assessment
  • Pharmacokinetic Modeling of Targeted Agents

By integrating real‑time imaging and biomarker analytics, trials aim to tailor dosing and reduce unnecessary exposure.