Pediatric Hematology Oncology PDF

PEDIATRIC HEMATOLOGY ONCOLOGY Claire Shearer, MA, RN | she/they | May 7 th, 2024  PEDIATRIC HEMATOLOGY ONCOLOGY Claire Shearer, MA, RN | she/they | May 7 th, 2024  PEDIATRIC HEMATOLOGY ONCOLOGY Claire Shearer, MA,...

PEDIATRIC HEMATOLOGY ONCOLOGY Claire Shearer, MA, RN | she/they | May 7 th, 2024  PEDIATRIC HEMATOLOGY ONCOLOGY Claire Shearer, MA, RN | she/they | May 7 th, 2024  PEDIATRIC HEMATOLOGY ONCOLOGY Claire Shearer, MA, RN | she/they | May 7 th, 2024  DISCLOSURE Anything marked with an * you will not be tested on I do not own the photos in this presentation No other disclosures PEDIATRIC HEMATOLOGY  PEDIATRIC HEMATOLOGY Iron Sickle Cell Hemophilia Thalassemia Deficiency Disease Anemia  HEMOPHILIA A group of bleeding disorders characterized by difficulty controlling bleeding and deficiencies in clotting factors Hemophilia A Hemophilia B  HEMOPHILIA Bleeding time is extended due to lack of a factor required for blood to clot Bleeding can be internal and/or external Sometimes bleeding disorders are recognized during infancy (i.e. circumcision, petechial/purpuric rashes, or unexpected bruising on children – especially under 6 months of age) “Those who don’t cruise rarely bruise” PURPURA & PETECHIAE  HEMOPHILIA A & B Both A and B are x-linked recessive disorders A: Deficiency of factor VIII, accounts for 80% of cases B: Deficiency of factor IX Tests for these may include PT/INR and aPTT (prolonged clotting times)  VON WILLEBRAND Autosomal inheritance - lack of Von Willebrand Factor protein Platelets unable to aggregate PTT and PT/INR may be WNL for these patients Tests for this may include Von Willebrand Factor Antigen and Von Willebrand Factor Activity  MANAGEMENT OF BLEEDING DISORDERS Management: Control/stop the bleed Typically avoid aspirin/NSAIDs (why?) sometimes used for significant joint inflammation in a person receiving factor replacement Minimize pokes when able ! Monitor for signs/sx of bleeding NSAIDs inhibit PLT aggregation  BLEEDING ASSESSMENTS Active bleeding (gums, epistaxis, hematuria, tarry stools) Hematomas/bruising Hemarthrosis as evidenced by joint pain, stiffness, warmth, swelling, loss of range of motion. Neuro assessment – headaches, neuro changes, slurred speech/lethargy Vital signs & pain assessment  MEDS FOR BLEEDING DISORDERS Meds: Antifibrinolytics like Amino-caproic acid (Amicar) Inhibits clot destruction DDAVP (Desmopressin) a synthetic form of vasopressin that increases plasma factor VIII Factor VIII or pooled plasma (IV infusion) Increases clotting factors May be prophylactic and/or used as a rescue Many families have their own factor at home for bleeding emergencies!  SICKLE CELL DISEASE World’s most common heritable blood disorder Protective factors against malaria Misperceptions persist in the US that SCD is exclusively a disease impacting Black-Americans RBCs, under hypoxic stress, polymerize into a “sickle” shape Median life expectancy of 42-47 in the United States (1994) Now nearly 95% of children with SCD will reach the age of 18 There are typically milder (HbSC and HbS beta thalessemia) and typically more severe (HbSS) forms of SCD Included in Newborn Screening (SCD and SCT) CDC; 5 FACTS YOU SHOULD K NOW  SICKLE CELL DISEASE CHARACTERISTICS Characterized by: Anemia (and associated symptoms) Vaso-Occlusive Pain Episodes (pain crises) Chronic Pain Acute and Chronic Organ Damage Mental Health Sequelae ! In severe acute Increased Susceptibility to Infection pain, people with SCD often face racist attitudes by clinicians  VASO-OC CLUSIVE EPISODES Characterized by SEVERE pain: “multiple fractures” and “broken bones and glass flowing through my body” ”like being stabbed repeatedly while having a migraine throughout your whole body” Possible Triggers: Dehydration Changes in temperature Stress Infection Ten Redefined High Altitude Artist: Hertz Nazaire  COMPLIC ATIONS OF SC D Acute Chest Syndrome: a life-threatening medical emergency, sickled cells block blood and oxygen from reaching the lungs. [AYA more common) This can occur from infection or when oxygenation to lung tissue is impaired. Signs and symptoms: fever, URI sx, infiltrate on CXR, cough, chest pain, low SpO2. Treatment: antibx, flluids, pain management, incentive spirometry Priapism: prolonged (>2 hrs), painful, and unwanted erection caused by sickled RBCs in the penis Repeated episodes over time or delayed treatment can cause permanent damage and erectile dysfunction Stroke: sickled RBCs get stuck in a blood vessel and block blood flow to the brain. Stroke risk increases 100-fold in children with SCD as compared to children without SCD About 10% of children with SCD will have a symptomatic stroke Recommended: Q2 years Trans Cranial Doppler (stroke risk, Moyamoya*, etc.)  COMPLIC ATIONS OF SC D Splenic Sequestration: occurs when sickled RBCs get trapped in the spleen and block blood flow, causing blood pooling and splenomegaly. Under functioning of the spleen (functional asplenia) in pediatric patients with SCD impairs immune function and puts them at a higher risk of blood stream infections and sepsis. Can progress to hypovolemic shock Fever & Infection: individuals with SCD are more likely to experience harmful infections (especially if their splenic function is impaired). Individuals with SCD should seek emergency care if they have a fever.  MANAGEMENT Hydration! Incentive Spirometry Collaborative Pain Management Pain assessment (function) PCAs Continuous infusions Multi-modal therapies (VR Goggles, warm packs, CCLS) ! Parents can Antibiotics be a great Oxygen resource for what works Administration of Blood Products best! Exchange transfusion  BIG TAKEAWAYS Believe and treat a patient’s pain Support oxygen requirements Prevent further hypoxic stress  BIG MEDIC ATIONS Prophylactic Penicillin (PenVK) Helps prevent pneumococcal infections in younger children and a- splenic children with SCD Pain Medications Tylenol and ibuprofen (mild – moderate) Opioids (moderate – severe) Hydroxyurea Increases fetal hemoglobin. Fetal hemoglobin helps keep RBCs round and circular and thus increases oxygen carrying capacity in the patient with SCD.  CU RE? Bone Marrow Transplants (BMT) (or HSCTs) are the only current FDA-approved cure for SCD, however, there are ongoing trials! Typically seen in younger children who have multiple complications. *A well-matched donor is needed for the best chance of a successful TXO* You will not be tested on specifics of BMTs CRISPR  *BONE MARROW TRANSPL ANT* *Bone Marrow Transplants (or Hematopoietic Stem Cell Transplants) Transfer of healthy bone marrow into a child with disease; the transplanted cells ideally then develop into functional cells Conditioning: Myeloablative & Immunosuppressive Some complications: GVHD Graft Failure  BMTS * Acute leukemia Multiple myeloma Adrenoleukodystrophy Myelodysplastic syndromes Aplastic anemia Neuroblastoma Bone marrow failure Non-Hodgkin's lymphoma syndromes Sickle Cell Disease Chronic leukemia SCIDs [Severe Combined Hemoglobinopathies Immuno-Deficiency] Hodgkin's lymphoma Thalassemia Immune deficiencies Inborn errors of metabolism  *BMT FACTS 70% of patients don’t have a fully-matched donor in their family. Their best hope for a cure is a blood stem cell transplant from an unrelated donor. May turn to Be The Match hoping to find an unrelated adult donor or umbilical cord blood unit. 3 – 12 months Post-BMT > revaccinate NO live vaccines  THALASSEMIA Reduced production of normal hemoglobin Genetically acquired Often diagnosed in childhood may present with severe anemia, jaundice, delayed growth, or skeletal deformities Beta-Thalassemia Major (Cooley’s – typically most severe): hemoglobin synthesis is reduced or entirely absent Treatment: Depends on severity (alpha or beta, major or minor) Some children receive PRBCs at regular intervals Iron Chelation  IRON DEFICIENCY ANEMIA The production of hemoglobin (Hgb) requires iron. Iron deficiency anemia usually results from an inadequate dietary supply of iron. Iron deficiency results in decreased Hgb levels. Adolescents are at risk due to poor diet, rapid growth, and menses. Risk factors: Excessive intake of cow’s milk Premature birth resulting in decreased iron stores Malabsorption disorders Poor dietary intake of iron  ASS ES SMENT Possible findings: Tachycardia Pallor Brittle, clubbed fingernails (chronic) Fatigue, irritability Systolic heart murmur Pica (cravings for non-nutritive substances i.e. ice/dirt) Labs may reveal decreased RBC/Hgb and Hct counts.  MANAGEMENT Iron supplementation in conjunction with vitamin C supplementation to increase absorption Give iron 1 hour before or 2 hours after milk, tea, or antacid to prevent decreased absorption. Give liquid iron through a straw to prevent staining of teeth. Expect kiddos taking iron supplements to have stools tarry green in color. Discuss with parents iron fortified cereals/formula Older kiddos: dried beans, lentils, peanut butter, leafy veggies, poultry and red meat Sometimes an RBC infusion, or an infusion of ferrous sulfate may be required RBCs given more slowly in individuals with severe anemia to prevent congestive heart failure and fluid volume overload IV Ferrous Sulfate is a very painful IV infusion and requires close monitoring during admin BLOOD TRANSFUSIONS  BLOOD PRODUC TS Cryoprecipitate: Help with clotting (factor). Plasma: Carries proteins, ions nutrients. Platelets: Help with clotting. Red blood cells: Help to oxygenate.  BLOOD PRODUCTS IN HEME ONC Oncology patients: Leuko-reduced CMV-negative Kids with Sickle Cell Disease: Hemoglobin S negative blood products Older kiddo’s type and cross may take longer to result because of repeat transfusions PEDIATRIC ONCOLOGY  ONCOLOGY LAB CONSIDERATIONS Absolute Neutrophil Count Neutrophils – first responders of the immune system Functional Neutropenia Blasts Neutrophils are not doing their job, even if ANC >500 Platelets Hematocrit/Hemoglobin Tumor Lysis Syndrome Labs Potassium, Uric Acid, Phosphate (PUP goes up!) Monitor renal function Typically administer allopurinol to mitigate TLS risk Decreases production of uric acid  MEDIASTINAL MASS Mediastinal mass More common in malignant lymphomas Risk compromising an airway/anatomy that is already small  SCIDs Osteosarcoma Rhabdomyosarcoma Neuroblastoma DIPG Wilm’s Tumor  SEVERE COMBINED IMMUNODEFICIENCY (SC IDS) A group of rare disorders caused by mutations in immune system development and function. Infants born with SCIDs appear healthy at birth, but are extremely immuno- compromised and highly susceptible to severe infections. David’s NASA-designed suit The condition is fatal without immune-restoring therapies (i.e. David Vetter circa 1976 Bone Marrow Transplant).  SEVERE COMBINED IMMUNODEFICIENCY (SC IDS) David Vetter, pictured, was born in 1971 and famously lived 12 years in a ”germ-free plastic bubble”. Curative therapies were still under investigation and development at that time. David’s NASA-designed suit Included in Newborn Screening David Vetter circa 1976  OSTEOSARCOMA The most common bone tumor found in pediatric patients. Typically develops after 10 years of age (more common during growth spurt period). Males are at a slightly increased risk. Most often found in long bones of the legs and arms. Two of the most common locations are the distal femur and the proximal tibia* rotation-plasty* Typical treatment includes chemotherapy and surgical excision of the tumor. *Rotationplasty  RHABDOMYOSARCOMA The most common soft tissue tumor of, these tumors can arise in varying anatomic locations and can resemble fat, fibrous tissue, and muscle. Two-thirds of cases are diagnosed in children under the age of 6. Treatment is typically multi-modal (surgical, chemotherapeutics, may include radiation, etc.) *Between 1975 and 2017, the 5-year relative survival rate for patients with rhabdomyosarcoma increased from 53% to 71% for children younger than 15 years and from 30% to 52% for adolescents aged 15 to 19 years. (NCI)  NEUROBLASTOMA Neuroblastoma is a rare pediatric cancer that affects the sympathetic nervous system in the adrenal glands, abdomen, and chest. *High-risk neuroblastoma can currently be treated with surgery, radiation, high-dose chemotherapy with autologous stem cell transplantation and monoclonal antibody therapy.* *Treatment typically lasts around 2 years.* Typically found in children 5 and under. Neuroblastoma is very rare in patients over 10 years old.  DIFFUSE INTRINSIC PONTINE GLIOMA A tumor impacting the pons, most commonly found in children About 10%-20% of all pediatric brain tumors Particularly challenging to treat because of the central location Multi-modal therapies are under experimentation, however, there is still no known cure for DIPG. Median survival: 10-11 months. These kiddos may go home with hospice/palliative care or may enroll in a trial  WILM’S TUMOR “Nephroblastoma” that occurs in the kidneys or the abdomen. The tumor is typically unilateral Most cases between ages 2 & 3 Requires surgical removal of the tumor/kidney “Do not palpate the abdomen” Once a Wilms tumor is suspected, subsequent abdominal examinations should be performed carefully. Vigorous palpation may rupture the renal capsule, resulting in tumor spillage, which increases the tumor stage and the need for more intensive therapy.  FEVER & NEUTROPENIA (F&N) Fever & Immunocompromised either a single temperature >39°C or two successive temperatures >38.4°C Neutropenia: Mild Neutropenia: ANC 1000 – 1500/microL Moderate Neutropenia: ANC 500 -1000 Severe neutropenia: ANC < 500 Parenteral antibiotics required These ranges vary institution to institution Classic signs of infection may be less evident in patients with neutropenia  INFEC TION RISK What might be some sites of infection risk in the pediatric oncology patient? Central Line (CLABSI) Mouth (oral hygiene is important) Diaper area (especially if higher risk of skin breakdown) Any area where they may have skin breakdown (nose pickers, if they like to pick scabs, constipation/difficulty passing stool, etc.) A common side effect of chemotherapy is mucositis – what does mucositis mean for infection risk?  SOCIAL CONSIDERATIONS Mental Health Sequelae these patients and pediatric cancer survivors may face Preservation of fertility in pediatric and adolescent patients with cancer (offering egg harvesting if time and age allow, offering sperm storing if age allows) How alopecia may impact a pediatric patient (developmental considerations of teenagers) Engaging children in their care and care decisions when age appropriate and plausible HAPPY STUDYING!