Our Pediatric Care Services specialize in providing advanced and compassionate treatment options for children diagnosed with various cancers and blood disorders.
What are the Treatments offered?
1. Pediatric Leukemias and Lymphomas
Disease: Pediatric leukemias, including Acute Lymphoblastic Leukemia (ALL) and Acute Myeloid Leukemia (AML), are the most common childhood cancers. Lymphomas, such as Hodgkin (HL) and Non-Hodgkin Lymphoma (NHL), arise from lymphatic tissues and are more common in adolescents.
Clinical Features: Leukemias present with fever, fatigue, pallor, bruising, recurrent infections, and bone pain due to marrow infiltration. Lymphomas often manifest as painless lymphadenopathy, fever, night sweats, weight loss, and mediastinal masses causing respiratory symptoms.
Diagnosis: Leukemias are diagnosed with blood tests (cytopenias, blasts), bone marrow biopsy, flow cytometry, and genetic analysis (e.g., BCR-ABL). Lymphomas require imaging (CT/PET scans) and lymph node biopsy for histopathology and staging.
Management: Chemotherapy forms the cornerstone of treatment. Targeted therapies like tyrosine kinase inhibitors (e.g., imatinib for ALL) and monoclonal antibodies (e.g., rituximab for NHL) improve outcomes. CAR T-cell therapy (e.g., tisagenlecleucel) has transformed care for relapsed ALL. HSCT offers curative potential in high-risk cases. Novel strategies include gene-edited CAR T-cells and immune checkpoint inhibitors for lymphomas.
2. Brain Tumors and Solid Tumors
Disease: Pediatric brain tumors (e.g., medulloblastoma, glioma, ependymoma) and extracranial solid tumors (e.g., neuroblastoma, wilms tumor, rhabdomyosarcoma, osteosarcoma) represent diverse malignancies. Neuroblastoma arises from neural crest cells and commonly involves the adrenal glands.
Clinical Features: Brain tumors present with headaches, vomiting, visual changes, seizures, or neurological deficits depending on location. Neuroblastoma may cause abdominal masses, bone pain, or paraneoplastic syndromes (e.g., hypertension, diarrhea). Wilms tumor presents with a painless abdominal mass, while osteosarcoma often causes bone pain and swelling.
Diagnosis: Brain tumors require MRI/CT, biopsy, and molecular profiling (e.g., MYC amplification in medulloblastoma). Neuroblastoma uses MIBG scans, bone marrow biopsy, and ALK mutation analysis. Other solid tumors are diagnosed via imaging and biopsy.
Management: Treatment includes surgery for resectable tumors, chemotherapy, and radiotherapy. Immunotherapy with anti-GD2 monoclonal antibodies (e.g., dinutuximab) has improved survival in high-risk neuroblastoma. Advances include ALK inhibitors (e.g., lorlatinib), tumor vaccines, and precision medicine targeting genetic mutations in other solid tumors like osteosarcoma.
3. Blood Disorders (Thalassemia, Sickle Cell Disease, and Hemophilia)
Disease: Thalassemia and Sickle Cell Disease (SCD) are genetic hemoglobinopathies, while hemophilia is caused by clotting factor deficiencies (Factor VIII in hemophilia A, Factor IX in hemophilia B).
Clinical Features: Thalassemia causes anemia, jaundice, splenomegaly, and growth retardation. SCD presents with pain crises, anemia, jaundice, stroke, and organ damage. Hemophilia causes prolonged bleeding, hemarthroses, and joint damage.
Diagnosis: Hemoglobinopathies are diagnosed with hemoglobin electrophoresis, genetic testing, and CBC. Hemophilia is identified by prolonged APTT, clotting factor assays, and genetic analysis. Regular screening for complications like iron overload (thalassemia) and organ damage (SCD) is essential.
Management: HSCT is curative for severe thalassemia and SCD. Gene therapy (e.g., lovotibeglogene autotemcel for SCD and beta-thalassemia) corrects genetic defects. For hemophilia, gene therapy (e.g., valoctocogene roxaparvovec) enables long-term factor production, reducing bleeding episodes and reliance on prophylactic infusions. Advanced approaches like CRISPR-based editing hold promise for durable cures.
