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Medical Conditions

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Wilms’ tumor / Nephroblastoma medical condition

Wilms’ tumor / Nephroblastoma

Wilms tumor, also called nephroblastoma, is a rare kidney cancer that mainly affects young children, most often between ages 3 and 4. It is the most common kidney cancer in children. After age 5, Wilms tumor becomes much less common, though it can rarely occur in older children and adults. In most cases, Wilms tumor affects only one kidney, but in some children it can occur in both kidneys at the same time. Thanks to major advances in diagnosis and treatment, the prognosis for Wilms tumor is generally very good, and most children can be cured with appropriate therapy. Symptoms Symptoms vary widely. Some children have no noticeable symptoms, while others may experience: A palpable mass or lump in the abdomen Abdominal swelling Abdominal pain Other possible symptoms include: Fever Blood in the urine Anemia (low red blood cell count) High blood pressure When to see a doctor Contact your child’s healthcare provider if you notice: A persistent abdominal mass or swelling Blood in the urine Unexplained pain or fever Wilms tumor is rare, and these symptoms are often caused by other conditions, but it’s important to investigate any concerns promptly. Causes The exact cause of Wilms tumor is not known. Cancer begins when cells develop changes (mutations) in their DNA. These changes cause kidney cells to grow and multiply uncontrollably, forming a tumor. In rare cases, inherited genetic changes passed from parents to children may increase risk. Risk factors Factors that increase the risk of Wilms tumor include: Race: Slightly higher risk in Black children; lower risk in Asian-American children Family history of Wilms tumor Certain congenital conditions are also associated with higher risk: Aniridia (partial or absent iris) Hemihypertrophy (one side of the body larger than the other) Wilms tumor may occur as part of rare genetic syndromes, such as: WAGR syndrome Denys–Drash syndrome Beckwith–Wiedemann syndrome Prevention Wilms tumor cannot be prevented. For children with known risk factors or associated genetic conditions, healthcare providers may recommend regular kidney ultrasounds. While screening does not prevent cancer, it can help detect Wilms tumor early, when treatment outcomes are best.

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Medulloblastoma medical condition

Medulloblastoma

Medulloblastoma (muh-DUL-oh-blas-TOE-muh) is a malignant (cancerous) brain tumor that begins in the cerebellum, the lower back part of the brain responsible for balance, coordination and movement. Medulloblastoma starts as a fast-growing mass of abnormal cells. These cells can spread within the brain and spinal cord through the fluid that surrounds them, called cerebrospinal fluid (CSF). Unlike many other cancers, medulloblastoma rarely spreads outside the brain and spinal cord. Although medulloblastoma can occur at any age, it is most common in young children. It is rare overall, but it is the most common malignant brain tumor in children. The risk is higher in families with certain inherited cancer-predisposition syndromes, such as Gorlin syndrome or Turcot syndrome. Symptoms Symptoms usually develop as the tumor grows or increases pressure inside the brain. Common signs and symptoms include: Headaches, often worse in the morning Nausea and vomiting Dizziness Double vision Poor coordination or clumsiness Unsteady walking Tiredness or weakness When to see a doctor Make an appointment with a healthcare professional if you or your child has symptoms that are persistent, worsening, or concerning. Causes The exact cause of medulloblastoma is not fully understood. Medulloblastoma develops when brain cells acquire changes in their DNA. DNA contains instructions that control how cells grow, divide and die. In healthy cells, these processes are tightly regulated. In cancer cells, DNA changes cause the cells to multiply rapidly and avoid normal cell death. As these abnormal cells accumulate, they form a tumor that can press on nearby brain structures, invade healthy tissue, and spread through the cerebrospinal fluid to other parts of the brain and spinal cord. Risk Factors Factors that may increase the risk of medulloblastoma include: Young age – most cases occur in children Inherited genetic syndromes that increase cancer risk, including: Fanconi anemia Gorlin syndrome Li-Fraumeni syndrome Rubinstein-Taybi syndrome Turcot syndrome Key Points to Know Medulloblastoma is aggressive but highly treatable, especially when diagnosed early Treatment often involves a combination of surgery, radiation therapy and chemotherapy Outcomes have improved significantly due to advances in pediatric neuro-oncology Early diagnosis, specialized care and long-term follow-up are essential for the best possible outcomes.

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Myelodysplastic Syndromes (MDS) medical condition

Myelodysplastic Syndromes (MDS)

Myelodysplastic syndromes (MDS) are a group of bone marrow disorders in which the marrow fails to produce sufficient healthy blood cells. These conditions arise from mutations in hematopoietic stem cells, leading to ineffective blood formation and the accumulation of immature or abnormal cells. As a result, patients may experience anemia, infections, and bleeding. Although the exact cause of these mutations is often unknown, MDS has been linked to advanced age, genetic predisposition, environmental exposures, and prior chemotherapy or radiation therapy. MDS is considered rare, with an estimated incidence of 4 per 100,000 people annually, though rates are significantly higher in individuals over 60. In some cases, MDS can progress to acute myeloid leukemia (AML). Types of Myelodysplastic Syndromes MDS is classified based on the number of affected blood cell lines, the presence of abnormal cells, and genetic findings: MDS with Single Lineage Dysplasia (MDS-SLD): One blood cell type is dysplastic, usually associated with cytopenia. MDS with Multilineage Dysplasia (MDS-MLD): Two or more blood cell lines are affected, often causing more severe symptoms. MDS with Ring Sideroblasts (MDS-RS): MDS-RS-SLD – ring sideroblasts with single lineage dysplasia MDS-RS-MLD – ring sideroblasts with multilineage dysplasia MDS with Excess Blasts (MDS-EB): MDS-EB1 – 5–9% blasts in blood or 5–10% in marrow MDS-EB2 – 10–19% blasts in blood or 10–20% in marrow MDS with isolated del(5q): Characterized by a chromosome 5 deletion, typically with anemia and normal or elevated platelets. MDS, Unclassifiable (MDS-U): Does not meet criteria for other categories. Each subtype carries a variable risk of progression to AML. Causes and Risk Factors Key risk factors include: Age (most cases occur after 60) Environmental exposures (benzene, pesticides, tobacco smoke) Prior cancer therapy (therapy-related MDS) Inherited bone marrow failure syndromes Rare familial genetic predisposition Lifestyle factors such as smoking and alcohol use Symptoms of MDS Symptoms reflect low or dysfunctional blood cell production: Anemia: fatigue, weakness, shortness of breath, pallor Leukopenia / neutropenia: recurrent or severe infections Thrombocytopenia: easy bruising, bleeding, prolonged clotting Severity ranges from mild to life-threatening and significantly impacts quality of life. Diagnosis Diagnosis requires a comprehensive hematologic evaluation: Complete blood count (CBC) Peripheral blood smear Bone marrow aspiration and biopsy Cytogenetic and molecular genetic testing Flow cytometry for cellular characterization These tests allow accurate classification, prognostication, and treatment planning. Treatment Options Treatment is personalized based on MDS risk level, age, health status, and goals of care. Lower-Risk MDS Supportive care (blood and platelet transfusions) Erythropoiesis-stimulating agents (ESAs) Iron chelation therapy Immunosuppressive therapy in selected cases Higher-Risk MDS Hypomethylating agents (azacitidine, decitabine) Chemotherapy Allogeneic stem cell transplantation (the only curative option) Participation in clinical trials Living with MDS Managing MDS involves addressing both physical and emotional challenges. Regular follow-up, symptom control, nutritional support, and psychological care are essential. Palliative and supportive services play a key role in maintaining quality of life. Expert Care at Sheba Medical Center The Hemato-Oncology Department at Sheba Medical Center specializes in the diagnosis and treatment of myelodysplastic syndromes, offering advanced molecular diagnostics, personalized therapies, and access to innovative clinical trials. Sheba’s multidisciplinary team is committed to delivering state-of-the-art care with compassion, recognizing that every MDS journey is unique.

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