Pediatric Blood & Bone Marrow Conditions We Treat
The Cancer Center at Nicklaus Children's Hospital provides state-of-the-art medical and surgical care to more pediatric cancer patients than any other facility in South Florida.
We are recognized for a multidisciplinary, holistic approach to care that places the child and family at the center of a highly coordinated network of specialists who work together to anticipate and meet the needs of the child and family throughout their treatment and follow-up care. When children enter the Center, they benefit from knowing that an entire team of pediatric specialists is putting their needs and the needs of their family first.
We have a comprehensive array of sub-specialists, programs and clinics covering the following conditions:
Acquired Aplastic Anemia
Please see Aplastic Anemia for further information.
Acute Lymphoblastic Leukemia (ALL)
There are several types of Leukemia. Acute lymphocytic leukemia (ALL), also called acute lymphoblastic leukemia, is the most common cancer and the most common leukemia affecting children. It is a cancer that affects the bone marrow and subsequently, the blood.
Learn more about Acute Lymphoblastic Leukemia (ALL).
Acute Myeloid Leukemia (AML)
Acute myeloid leukemia is a leukemia that starts in the bone marrow (the tissue inside the bone where blood is made) and results in the creation of immature white blood cells.
Learn more about Acute Myeloid Leukemia (AML).
An Adamantinoma is a rare slow growing type of cancerous bone tumor that occurs most often in boys and young men, most frequently found in the shin bone, and jaw bone.
Learn more about Adamantinoma.
Alpha thalassemia is an abnormality in the production of alpha chains which causes the red cells to more easily breakdown.
Learn more about Alpha Thalassemia.
Alpha Thalassemia Silent Carrier
Thalassemia is a medical condition in which the body makes less hemoglobin than usual. It causes anemia. There are four genes that contribute to the making of hemoglobin.
Learn more about Alpha Thalassemia Silent Carrier.
Alveolar Soft Part Sarcoma
Alveolar soft part sarcoma, or ASPS, is a very rare cancer that grows slowly from the connective tissue that usually occurs in children and young adults.
Learn more about Alveolar Soft Part Sarcoma.
Astrocytes cells are a diverse group of cells which play many roles in the brain, but particularly form the physical and physiological supportive system for the brain’s neurons. Astrocytomas are tumors that grow from these cells and make up almost 50% of childhood brain tumors, frequently occurring in children between 5-9 years of age.
Learn more about Anaplastic Astrocytoma.
An Angiofibroma is a rare benign tumor that occurs most frequently in young boys/adolescents, and grows in the nasal cavity.
Learn more about Angiofibroma.
Aplastic Anemia (AA)
When the bone marrow doesn’t produce enough blood cells, this condition is known as aplastic anemia.
Learn more about Aplastic Anemia (AA).
An Astrocytoma is a form of brain tumor (benign or cancerous), that develops in the cells that form the supportive tissue of the brain.
Learn more about Astrocytoma.
Atypical Teratoid Rhabdoid Tumor
An atypical teratoid rhabdoid tumor, is a fairly rare aggressive tumor usually diagnosed in young children that forms in the tissues of the Central Nervous System.
Learn more about Atypical Teratoid Rhabdoid Tumor.
Thalassemia is a medical condition in which the body makes less hemoglobin than usual. It causes anemia. There are two types of thalassemia, alpha and beta.
Learn more about Beta Thalassemia.
Beta Thalassemia Intermedia
Thalassemia is a medical condition in which the body makes less hemoglobin than usual. It causes anemia. There are two types of thalassemia, alpha and beta.
Learn more about Beta Thalassemia Intermedia.
Beta Thalassemia Major
Beta thalassemia is an abnormality in the production of beta chains which causes the red cells to more easily breakdown resulting in mild to severe anemia.
Learn more about Beta Thalassemia Major.
Beta Thalassemia Trait (Minor)
Thalassemia is a medical condition in which the body makes less hemoglobin than usual. It causes anemia. There are two types of thalassemia, alpha and beta.
Learn more about Beta Thalassemia Trait (Minor).
Bone Tumors and Cysts
Bone tumors and cysts are abnormal growths that can occur in the bones.
Learn more about Bone Tumors and Cysts.
The term brain tumor refers to an unusual growth of tissue seen in the brain irrespective of the nature and cause of the growth.
Learn more about Brain Tumors.
Lymphomas are cancers classified mostly according to the type of white cell involved. Burkitt’s lymphoma starts in B-lymphocytes, so-called because they mature in the bone marrow of many bones.
Learn more about Burkitt's Lymphoma.
If an abnormal tissue grows in the heart it is known as a primary cardiac tumor.
Learn more about Cardiac Tumor.
CDKL5 (Cyclin-Dependent Kinase-Like 5) is a gene located on the X-chromosome. The CDKL5 gene is required to function normally for a normal brain to develop.
Learn more about CDKL5 Disorder.
Chondroblastoma is a rare type of cartilage-producing benign tumor in children that often occurs in the ends of the long bones of the arms and legs.
Learn more about Chondroblastoma.
Chondrosarcoma is a form of cancer that can develop in the bones and soft tissues of the body, usually in people over 20 years of age. Mostly they begin in the cells in the joints that produce cartilage.
Learn more about Chondrosarcoma.
Choroid Plexus Brain Tumor
The choroid plexus is the tissue that lies in the cavities of the brain (called ventricles and there are four of them) that creates the fluid (cerebrospinal fluid) which surrounds and cushions the brain and spinal cord.
Learn more about Choroid Plexus Brain Tumor.
Chronic Granulomatous Disease
Chronic granulomatous disease (CGD) is the disorder that develops when phagocyte (one of the body's blood cells) doesn’t function properly.
Learn more about Chronic Granulomatous Disease.
Chronic Myelogenous Leukemia (CML)
Chronic myelogenous leukemia is a rare cancer of the blood in children where over time, too many immature white blood cells form because of an abnormality in the normal maturation process of the cell.
Learn more about Chronic Myelogenous Leukemia (CML).
Congenital Amegakaryocytic Thrombocytopenia
CAT is a rare disorder (two types) found in infants where there are very few megakaryocytes and platelets in bone marrow, and while initially the bone marrow produces red and white cells this eventually decreases /stops so the baby/child has a lack of platelets, red cells and white cells.
Learn more about Congenital Amegakaryocytic Thrombocytopenia.
Congenital anemia, including Fanconi anemia, is a group of inherited diseases that affect the bone marrow, decreasing production of all types of blood cells.
Learn more about Congenital Anemia.
A craniopharyngioma is a non-cancerous brain tumor that develops from cells present during early brain development, and in childhood frequently presents between the ages of 5-14 years.
Learn more about Craniopharyngioma.
Children with cyclic neutropenia have a fairly non-serious disease (benign) where their white blood cell count becomes low for 3-5 days, every 14-35 days.
Learn more about Cyclic Neutropenia.
A desmoid tumor in one that frequently arises in muscles (often abdominal wall and shoulder -legs and arms also may be affected), however can also come from tissues, tendons, and ligaments.
Learn more about Desmoid Tumor.
Diamond-Blackman anemia is a rare disorder of the bone marrow which fails to produce enough new red blood cells.
Learn more about Diamond-Blackfan Anemia.
Differentiated Thyroid Cancer
Thyroid cancer is cancer that impacts the thyroid gland, a butterfly-shaped gland located in the neck. The most common types of thyroid cancers are known as differentiated thyroid cancers.
Learn more about Differentiated Thyroid Cancer.
Diffuse Intrinsic Pontine Glioma (DIPG)
Glial tissue are cells of the brain that protects and supports the neurons. Tumors that start in the glial tissue at the base of the brain in the brainstem area (which controls breathing, heart rate and blood pressure plus other functions) called the Pons, just above the back of the neck, are called diffuse intrinsic pontine gliomas.
Learn more about Diffuse Intrinsic Pontine Glioma (DIPG).
Dysembryoplastic Neuroepithelial Tumor
Dysembryoplastic neuroepithelial tumors are rare, slow growing, non-spreading growths, found in children and teens, that develop from different types of abnormal central nervous system brain cells.
Learn more about Dysembryoplastic Neuroepithelial Tumor.
Dyskeratosis congenita is a rare form of bone marrow failure, with associated skin/nail abnormalities, and thickened white patches in the mouth.
Learn more about Dyskeratosis congenita.
Embryonal Brain Tumors
Embryonic (fetal) cells are a type of brain cell that remains in the brain after birth and while embryonic tumors can occur at any age they most often happen in babies and young children.
Learn more about Embryonal Brain Tumors.
Enchondromas are non-cancerous (usually) cartilage tumors that are found in the marrow of a bone (frequently the small bones of the hand, but can occur in the long bones of thigh, shin and upper arm).
Learn more about Enchondroma.
Ependymal cells form the lining of the ventricles (fluid containing spaces) in the brain and the central canal of the spinal cord. Tumors that develop from these cells, (fairly rarely, though 6 percent to 12 percent of childhood brain tumors) are called ependymomas. The average age at diagnosis is between 2 and 4 years.
Learn more about Ependymoma.
Epithelioid hemangioendothelioma is a rare vascular tumor of young adults that starts in the cells lining the blood vessels of soft-tissue, bone and body organs- commonly the liver, lungs and bone.
Learn more about Epithelioid Hemangioendothelioma.
Ewing sarcoma is a rare type of cancer that usually develops in the bones or soft tissue around the bones (cartilage/nerves) of children 10-20 years of age.
Learn more about Ewing sarcoma.
Fanconi anemia is a rare inherited disorder that involves a wide spectrum of abnormalities involving bone, ears, kidneys, other physical abnormalities. Some of the patients may dvelop bone marrow failure or leukemia. In addition, patients with Fanconi are at a very high risk of developing multiple cancers in their lifetime.
Learn more about Fanconi Anemia.
Fibrosarcoma is a rare form of soft tissue cancer that occurs in infants and children 10-15 years of age, that starts in the fibrous tissue, of the body but most commonly in the tendons at the ends of the limbs.
Learn more about Fibrosarcoma.
Please see Dysembryoplastic Neuroepithelial Tumor for further information.
Germinoma of the Central Nervous system
A germinoma is a rare form of cancer that is most often found in the brain of children between the ages of 10 and 19 years.
Learn more about Germinoma of the Central Nervous system.
Giant Cell Tumor
A giant cell tumor is a non-cancerous tumor (benign) that grows at the ends of the body’s long bones.
Learn more about Giant Cell Tumor.
Glioblastoma multiforme are high-grade 1V, aggressively growing, cancerous glial tumors which infiltrate into healthy adjacent brain tissue, frequently occurring in both boys and girls aged 5-10 years.
Learn more about Glioblastoma Multiforme.
Glioma is a form of cancer that develops from glial cells of the brain - those cells which support and nourish the neurons.
Learn more about Glioma.
Astrocytes are star-shaped cells that are part of a glial network of supportive and nourishing tissue within the brain. One rare form of malignant tumor that grows and infiltrates aggressively from these cells and which spreads over the top and throughout the brain is known as gliomatosis cerebri.
Learn more about Gliomatosis Cerebri.
Head and Neck Tumors
Head and neck growths, tumors or masses in children are usually benign (non-cancerous), but can be malignant (a cancer which spreads).
Learn more about Head and Neck Tumors.
If a tumor, or a foreign growth of tissue, develops in the heart, it is known as a heart or cardiac tumor. Tumors can develop on the heart tissue (primary, or myxoma) or start elsewhere in the body and spread to the heart (secondary). Some cause cancer (malignant), and some do not (benign).
Learn more about Heart Tumors.
A hemangiopericytoma is a very rare soft tissue mass due to overgrowth of capillaries that is almost always benign.
Learn more about Hemangiopericytoma.
Hemoglobin is the protein in the blood that carries oxygen from the lungs throughout the body. One abnormal form of hemoglobin is known as hemoglobin E.
Learn more about Hemoglobin E.
Anemia is a disorder in which the body doesn’t have enough red blood cells. Hemolysis is a method whereby red blood cells are broken down and destroyed.
Learn more about Hemolytic Anemia.
Hemolytic disease of the newborn is a condition that occurs when a mother’s blood type is not compatible with her unborn fetus.
Learn more about Hemolytic Disease.
The disease is an extreme example of immune system dysfunction, where the body’s own immune system begins to attack important tissues and organs such as the brain, liver and bone marrow.
Learn more about Hemophagocytic Lymphohistiocytosis.
Hemophilia is a medical condition in which children bleed longer than normal because their blood doesn't clot properly due to a lack of the proteins known as clotting factors.
Learn more about Hemophilia.
Hemophilia is a disease in which the blood is missing a component that makes it clot properly. The result is that the individual with hemophilia can bleed easily, bleed excessively or not stop bleeding once they start. In the case of hemophilia A, or classic hemophilia, the blood is deficient in factor VIII.
Learn more about Hemophilia A.
Hemophilia is a disease in which the blood is missing a component that makes it clot properly. The result is that the individual with hemophilia can bleed easily, bleed excessively or not stop bleeding once they start, among other problems. In the case of hemophilia B, the blood is deficient in factor IX.
Learn more about Hemophilia B.
Henoch-Schonlein purpura is a common vasculitis (inflammation and bleeding of blood vessels) of the small blood vessels of the skin, mucous membranes, joints, intestines and kidneys in children, between 2-6 years of age (slightly more common in boys and in Whites and Asians).
Learn more about Henoch-Schonlein Purpura.
A hepatoblastoma is a rare tumor (cancerous- spreads) that grows from the cells of the liver.
Learn more about Hepatoblastoma.
Hereditary Paraganglioma-Pheochromocytoma Syndrome
Hereditary paraganglioma-pheochromocytoma syndrome is a genetic condition that’s passed down through families. Individuals with the condition are more likely to develop two rare forms of cancer. One is known as paragangliomas, which are tumors on the spine or lower skull.
Learn more about Hereditary Paraganglioma-Pheochromocytoma Syndrome.
Hgb C Disease
Hemoglobin is the protein in the blood that carries oxygen from the lungs throughout the body. One abnormal form of hemoglobin is known as hemoglobin C, and it can cause red blood cells to break down earlier than normal, which leads to anemia.
Learn more about Hgb C Disease.
Hgb C Trait
Hemoglobin is the protein in the blood that carries oxygen from the lungs throughout the body. One abnormal form of hemoglobin is known as hemoglobin C.
Learn more about Hgb C Trait.
Hgb E Disease
Hgb E disease is a mild disorder, and people who have it may have no symptoms or mild anemia.
Learn more about Hgb E Disease.
Hgb E Trait
Hemoglobin is the protein in the blood that carries oxygen from the lungs throughout the body. Hemoglobin E trait affects the hemoglobin in the red blood cells but is a mild disorder.
Learn more about Hgb E Trait.
Hgb E/B + Thalassemia
Thalassemia is a medical condition in which the body makes less hemoglobin than usual. It causes anemia.
Learn more about Hgb E/B + Thalassemia.
Hgb E/B 0 Thalassemia
Hemoglobin B and E are abnormal forms of hemoglobin. Is a red blood cell disorder found on the newborn screening by presence of fetal hemoglobin (F), hemoglobin E and the absence of hemoglobin A (normal Hemoglobin).
Learn more about Hgb E/B 0 Thalassemia.
Hgb H Constant Spring
Hgb H disease is a form of alpha thalassemia, a medical condition in which the body makes less hemoglobin than usual. It causes anemia. There are four genes that contribute to the making of hemoglobin.
Learn more about Hgb H Constant Spring.
Hgb H Disease
Hemoglobin H disease is a form of alpha thalassemia, a medical condition in which the body makes less hemoglobin than usual.
Learn more about Hgb H Disease.
Lymphoma is a type of cancer that affects the body’s lymphatic system (immune system).
Learn more about Hodgkin Lymphoma.
Hydrops fetalis is an abnormal collection of fluid in at least two different organ spaces (like the skin, abdomen, around the heart and/or lungs) causing massive swelling.
Learn more about Hydrops Fetalis.
Idiopathic Thrombocytopenic Purpura (ITP)
"Idiopathic" means cause unknown. "Thrombocytopenia" means a low platelet count. Platelets are blood cells that help blood clot thereby stopping bleeding. "Purpura" is the purple color of the skin seen when bleeding into the skin occurs.
Learn more about Idiopathic Thrombocytopenic Purpura (ITP).
Immune Deficiency Syndromes
The body’s immune system primarily defends one’s body against infections like bacteria, viruses and parasites. There are two broad categories of immune deficiency - those that one is born with (congenital), and those that are acquired after birth (secondary). Immune deficiency syndrome refers to a broad range of medical disorders that prevent your body from protecting itself from illnesses such as viruses and bacteria. There are a number of different types of congenital and acquired immune deficiency syndromes that can impact the body in a variety of ways.
Learn more about Immune Deficiency Syndromes.
Inborn Errors of Metabolism
When a baby has trouble digesting certain foods and turning them into energy, it
could be due to inborn errors of metabolism.
Learn more about Inborn Errors of Metabolism.
Juvenile Myelomonocytic Leukemia
JMML is a rare, serious chronic form of cancer of the blood in children (frequently boys), aged less than 4 years.
Learn more about Juvenile Myelomonocytic Leukemia.
Juvenile Polyposis Syndrome
Juvenile polyposis syndrome is a disease that is known for causing polyps different areas of the gastrointestinal tract, most commonly the colon. Polyps are clusters of cells that form on the lining of the GI tract.
Learn more about Juvenile Polyposis Syndrome.
Kostmann's syndrome is a disease of the bone marrow where children are born without a type of white blood cell
Learn more about Kostmann’s Syndrome.
Langerhans Cell Histiocytosis
Langerhans cell histiocytosis (LCH) is a rare disease of children in which a certain immature normal white blood type grows uncontrollably...
Learn more about Langerhans Cell Histiocytosis.
Large Cell Lymphoma
Large cell lymphoma is a common, fast growing group of blood cancers that affect one cell type of white blood cell called lymphocytes.
Learn more about Large Cell Lymphoma.
Leiomyosarcoma is a very rare type of soft tissue cancer that affects the smooth muscles of the body.
Learn more about Leiomyosarcoma.
Li-Fraumeni syndrome is a genetic disorder that increases the risk of getting certain forms of cancer in children and adults.
Learn more about Li-Fraumeni Syndrome.
Liposarcoma is a cancer of fat cells. It can occur anywhere in the body, but is commonly found in the abdomen, thigh or shoulder area.
Learn more about Liposarcoma.
Liver tumors are abnormal growths within the liver that can be either benign (non-spreading ) or cancerous.
Learn more about Liver Tumors.
Glioma is a form of cancer that develops in the glial cells of the brain.
Learn more about Low-Grade Gliomas.
Lymphomas are a type of cancer involving lymphocytes, which are cells within the immune system that help the body fight off infections.
Learn more about Lymphomas.
Malignant Fibrous Histiocytoma
Malignant fibrous histiocytoma is a rare form of cancer in children known as a soft tissue sarcoma. These are tumors that affect muscle, bone or cartilage, or tissues that support/connect other organs.
Learn more about Malignant Fibrous Histiocytoma.
Malignant Rhabdoid Tumor
A malignant rhabdoid tumor is a rare fast growing childhood cancer that commonly occurs in infants and young children (average age 15 months).
Learn more about Malignant Rhabdoid Tumor.
Medullary Thyroid Cancer
Medullary thyroid cancer (MTC) is a form of cancer that originates in a particular cell in the thyroid gland.
Learn more about Medullary Thyroid Cancer.
Of cancerous brain tumors that can affect children, medulloblastoma is the most common. They represent about 20 percent if childhood brain tumors, particularly in children between the ages of 3 and 8 years, with boys affected more than girls.
Learn more about Medulloblastoma.
Megaloblastic anemia is a type of anemia where the bone marrow produces fewer and abnormally large, oval shaped (instead of round/disk-like) red blood cells, with underdeveloped inside contents (hemoglobin).
Learn more about Megaloblastic Anemia.
Megaloblastic Pernicious Anemia
Please see Megaloblastic Anemia for further information.
Melanocytes are the cells in the body’s skin that produce melanin. Melanin is the pigment that gives skin its color. When cancer develops in the melanocytes, it is known as melanoma. Of the many forms of skin cancer, melanoma is generally regarded as the most dangerous.
Learn more about Melanoma.
A meningioma is a relatively uncommon childhood tumor that grows from the middle of the three membranes layers that cover the brain and spinal cord.
Learn more about Meningioma.
Mental Health (Sickle Cell Disease)
Mental health (sickle cell disease) can manifest itself in several different ways, including anxiety, depression and even psychosis over time.
Learn more about Mental Health (Sickle Cell Disease).
Metabolic Storage Diseases
Metabolic storage diseases are a fairly large group of rare genetic inherited disorders in which specific enzymes of a child's cell are insufficient. Enzymes help break down certain materials within the cell for proper cell function.
Learn more about Metabolic Storage Diseases.
Multiple Endocrine Neoplasia Type 1
Multiple endocrine neoplasia type 1 is a disease that affects several glands throughout the body, including the thyroid, parathyroid and pituitary gland. It causes tumors in these glands and also imbalances in the hormones that the glands produce. The pancreas and small intestine are also impacted by the disease.
Learn more about Multiple Endocrine Neoplasia Type 1.
Multiple Endocrine Neoplasia Type 2
Multiple endocrine neoplasia type 2 is a disease that affects several glands throughout the body, including the thyroid, parathyroid and adrenal gland. It causes tumors in these glands and also imbalances in the hormones that the glands produce. It differs from multiple endocrine neoplasia type 1 based on the genetic mutation that causes it, and some of the glands that it impacts.
Learn more about Multiple Endocrine Neoplasia Type 2.
Myelodysplastic syndrome (MDS) is referred to a group of different types of cancer. But they all impact the bone marrow and prevent it from developing mature, healthy blood cells. Patients have low blood counts and present like Aplastic Anemia.
Learn more about Myelodysplastic Syndrome.
Neuroblastoma is a form of cancer that primarily affects children. Among infants, it ranks as the most common form of cancer. The cancer forms in nerve cells called neuroblasts. It usually occurs in the adrenal glands above the kidneys, but can also start anyway along the nerves in the back.
Learn more about Neuroblastoma.
Neurofibrosarcoma is a rare type of malignant cancer in children that develops in the cells of the sheath of the arms and legs, spreading along the nerves.
Learn more about Neurofibrosarcoma.
Non-Hodgkin lymphoma is an uncommon type of cancer in children. It starts in the body’s lymphatic system which are part of the body’s defenses against infections.
Learn more about Non-Hodgkin lymphoma.
A Non-Ossifying Fibroma (NOF) is a common, benign fibrous tissue (like a scar) tumor in boys and girls that is usually found in the thigh (femur) or shin (tibia) bones (but can occur in other bones).
Learn more about Non-Ossifying Fibroma.
Ocular tumors are any type of tumor that impacts the eyeball or the surrounding area. They can be benign or malignant.
Learn more about Ocular Tumors.
Odontogenic tumor is the medical term for a growth or cyst that affects the jaw.
Learn more about Odontogenic Tumors.
Oligodendroglioma is a type of brain tumor that affects the glial tissue of the brain.
Learn more about Oligodendroglioma.
Optic Pathway Glioma
Glioma is a specific type of slow growing tumor that starts in one of the cells of the brain or spinal cord. When this tumor grows on or around the visual system it is known as an optic pathway glioma.
Learn more about Optic Pathway Glioma.
Osteopetrosis keeps building up bone without the ability to remove the excess. Therefore people with the condition have bones that are very dense and more likely to fracture than normal bones.
Learn more about Osteopetrosis.
Osteosarcoma is a very common type of bone cancer that affects older children and adolescents. It usually starts in the areas of long bones that are still growing, like at the ends of the thigh, shin or upper arm.
Learn more about Osteosarcoma.
The palate is another word for the roof of the mouth. When abnormal growths of cells known as tumors impact the palate, these are commonly known as palatal tumors.
Learn more about Palatal Tumors.
Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria is a rare, dangerous, acquired blood disorder during which unpredictable episodes of blood red cell breakdown occurs, the bone marrow stops making new blood cells and blood clots.
Learn more about Paroxysmal Nocturnal Hemoglobinuria.
Peutz-Jeghers syndrome is a genetic disorder that causes affected individuals to develop polyps in their intestines. Polyps are small growths of tissue that are typically non-cancerous, but they can increase the risk of cancer developing over time. Other symptoms can also occur with Peutz-Jeghers syndrome.
Learn more about Peutz-Jeghers Syndrome.
An astrocytoma is a type of brain tumor glioma, and pilocytic astrocytomas are a family of slow-growing, mostly noncancerous (non spreading-grades 1 and 11) tumors from glial cells.
Learn more about Pilocytic Astrocytoma.
Platelet Function Disorders
Platelets are a small type of cell that circulates in the blood that helps the blood clot properly. They do this by clumping together and plugging up holes (injuries) in blood vessels. There are normally between 150,000 - 350,000 of them. A child may have less than the normal number of platelets (thrombocytopenia), or a normal number of platelets than don't function properly (thrombasthenia).
Learn more about Platelet Function Disorders.
Pleomorphic xanthoastrocytoma is a rare brain tumor that grows in the upper parts of the brain (cerebral hemispheres) from brain cells called astrocytes (a type of glioma), which form the brain’s supportive and nutritional network.
Learn more about Pleomorphic Xanthoastrocytoma.
Polycythemia is a rare and serious blood disease that causes the bone marrow to produce too many red blood cells (adjusted for race, age, sex and altitude) to circulate in the blood stream.
Learn more about Polycythemia.
Primitive Neuroectodermal Tumors (PNET)
Primitive neuroectodermal tumors are a group of cancers (malignant) that start in nerve cells formed in the fetus during early development that haven't developed the way they should have.
Learn more about Primitive Neuroectodermal Tumors (PNET).
Rare Bleeding Disorders
Rare bleeding disorders refer to many uncommon types of medical conditions such as hemophilia and others that impact the body’s ability to stop bleeding, or clot blood properly.
Learn more about Rare Bleeding Disorders.
Red Blood Cell Disorders
Red blood cells (RBC’s; erythrocytes) are the component of blood that carries oxygen from the lungs to all parts of the body. There are a large number of hereditary (congenital) and acquired disorders that affect the production, number, shape, hemoglobin content and friability of red blood cells.
Learn more about Red Blood Cell Disorders.
Relapsed or Refractory Neuroblastoma
Neuroblastoma is the most common solid cancerous tumor found outside the brain in children.
Learn more about Relapsed or Refractory Neuroblastoma.
The retina is the tissue at the back of the eyes that allows children to see both light and color. When cancer develops in the retina, this is known as a retinoblastoma. This type of cancer frequently affects very young children, before the age of 5.
Learn more about Retinoblastomas.
Rhabdoid Tumor Predisposition Syndrome
Rhabdoid tumors are abnormal growths that frequently affect the brain, kidneys or spinal cord, among other parts of the body.
Learn more about Rhabdoid Tumor Predisposition Syndrome.
Rhabdomyosarcoma is a type of soft tissue cancer that can affect any of the skeletal muscles, or voluntary muscles, of the body occurring in boys more often than girls.
Learn more about Rhabdomyosarcoma.
A sacrococcygeal teratoma is a tumor containing a number of different tissues (fat, bone, nerves etc.) that is present at birth in the tailbone (coccyx) of the newborn baby.
Learn more about Sacrococcygeal Teratoma.
Sarcomas are rare kinds of cancers that affect the body’s soft tissues. They are given different names depending on the type of cell or tissue involved including muscles, tendons, bone, fat, etc.
Learn more about Sarcomas.
Severe Combined Immune Deficiency
Severe combined immune deficiency is a rare genetic birth defect that doesn’t allow the body to develop an immune system, which fights all types of infections.
Learn more about Severe Combined Immune Deficiency.
Shwachman-Diamond syndrome is a rare complex disorder that affects a child's bone marrow, pancreas and bones (and sometimes other parts of the body).
Learn more about Shwachman-Diamond Syndrome.
Sickle Beta + Thalassemia
Sickle beta + thalassemia is a disease that cause a mild form of sickle cell anemia. This causes the body’s hemoglobin, or red blood cells, to take on a sickle shape and not flow through the blood vessels as smoothly.
Learn more about Sickle Beta + Thalassemia.
Sickle Beta 0 Thalassemia
Sickle beta 0 thalassemia is an inherited disease. It causes the red blood cells to have abnormal hemoglobin.
Learn more about Sickle Beta 0 Thalassemia.
Sickle Cell C Disease
Hemoglobin is a protein in blood that carries oxygen from the lungs throughout the body.
Learn more about Sickle Cell C Disease.
Sickle Cell Disease
Sickle cell disease is a group of familial red blood cell disorders. Sickle cell disease causes the red blood cells to be oddly shaped, and have difficulty flowing through the blood vessels properly which causes them to break up easily resulting in anemia and damage to the organs.
Learn more about Sickle Cell Disease.
Sickle Cell Trait
Sickle cell trait (SCT) is not a disease, but having it means that a person is a carrier that has inherited the sickle cell gene from one of the parents.
Learn more about Sickle Cell Trait.
SO Arab - Sickle Cell Disease
Learn more about SO Arab - Sickle Cell Disease.
Soft Tissue Sarcomas
Soft tissue sarcomas are cancers are rare kinds of cancers that affect the body’s soft tissues.
Learn more about Soft Tissue Sarcomas.
Spider angioma/s are one or more common benign abnormal clusters of blood vessels that are often visible on the skin of the face, neck, upper arms, hands, fingers and trunk.
Learn more about Spider Angiomas.
Spinal Cord Tumors
If a tumor grows within (~ 10%), in the tissues around, or spreads from a different site to the spinal cord region, it is known as a spinal cord tumor.
Learn more about Spinal Cord Tumors.
The synovial membrane is a thin layer of tissue that lines the part of a joint that acts as a shock absorber between joints of bones. When the synovium thickens and fragments break off, the pieces may grow, calcify (become bone-like nodules) and become loose in the joint. This is synovial chondromatosis. Usually this process is benign (non-cancerous), however rarely, SOC may be become malignant.
Learn more about Synovial Chondromatosis.
Synovial sarcomas are a rare form of slow growing soft tissue cancer that commonly forms around the joints in the arms or legs in adolescent or young adults.
Learn more about Synovial sarcoma.
A tectal glioma ( from a type of glial cell that nourishes and supports other brain cells) is a slow growing, generally benign (non spreading), brain tumor in children 3-16 years of age, situated in the upper portion or roof of the brain stem ( this area of the brain controls important body functions like breathing, heart rate, and blood pressure).
Learn more about Tectal Gliomas.
A teratoma is a fairly common congenital (develops before birth) type of solid tumor that contains normal looking tissues like hair, teeth and bone (and very rarely whole organs like, for example eyes).
Learn more about Teratoma.
Testicular tumors are growths on the testicle. They occur in infant boys < 2 years of age (when they are usually benign - non-cancerous and don't spread) and after boys enter puberty (when most are malignant; cancerous and can spread).
Learn more about Testicular Tumors.
Thalassemia is a blood disorder in which the body produces hemoglobin that doesn’t function properly.
Learn more about Thalassemia.
Thalassemia is a medical condition in which the body makes less hemoglobin than usual. It causes anemia. There are four genes that contribute to the making of hemoglobin
Learn more about Thalassemia Trait.
Platelets (thrombocytes) are cells produced in the bone marrow that help the blood to clot by clumping together and forming a plug, at the site of a damaged blood vessel. Thrombocytopenia is the condition where there is a low platelet count (less than 150,000/microL) and this results in bleeding (hemorrhage) because the blood doesn't clot properly.
Learn more about Thrombocytopenia.
Thrombocytopenia-Absent Radius Syndrome
Thrombocytopenia-absent radius syndrome, or TAR syndrome, is a rare disorder present at birth that is characterized by the absence of a bone called the radius in both forearms, as well as thrombocytopenia (low platelet count) and short stature.
Learn more about Thrombocytopenia-Absent Radius Syndrome.
Thrombophilia is an abnormality of the normal blood clotting mechanisms.
Learn more about Thrombophilia.
When a blood clot forms in a blood vessel, the process is known as thrombosis.
Learn more about Thrombosis.
Please see Thyroid Nodules for further information.
The thyroid gland is a butterfly-shaped organ located in the front of the base of the neck that produces hormones that regulate many important functions of the body (e.g. breathing, heart rate, brain and nerve function, body weight, temperature muscle strength and many others). A thyroid nodule (or lump) is a fairly rare unusual firm growth of cells that occur within the thyroid gland and there are several types. While thyroid nodules are frequently benign in children (non-cancerous) they are more likely to be cancerous than they are in adults.
Learn more about Thyroid Nodules.
Vascular anomalies is a broad term that refers to blood vessels (or lymphatic vessels) that grow or form in unusual way. Many birthmarks are due to vascular anomalies, but they can occur for other reasons, as well.
Learn more about Vascular Anomalies.
Vitamin K deficiency bleeding
Vitamin k plays an important role in blood clotting (coagulation); particularly being associated with coagulation factors V11, 1X, and X. When there is a lack of Vit k blood doesn't clot and bleeding results.
Learn more about Vitamin K deficiency bleeding.
Von Willebrand Disease
VMD is a bleeding disorder. Children with Von Willebrand disease (VWD) have a defect or are missing a blood clotting protein called the Von Willebrand factor.
Learn more about Von Willebrand Disease.
White Blood Cell Disorders
White blood cells (also called leukocytes) are the cells in your blood that help your body fight off infection.
Learn more about White Blood Cell Disorders.
Wilms tumor is a rare type of cancer that starts in the kidney and occurs most often in children ages 3 or 4.
Learn more about Wilms’ tumor.