• Starting dose: 15 mg/kg/day, increase gradually by 5 mg/kg/day while monitoring for toxicity.
• Toxicity manifested by falls in neutrophil count to <2500/mm
3
or platelet count <80,000/mm
3
.
f. Pain medications: acetaminophen or nonsteroidal anti-inflammatory drugs (NSAIDs) (mild pain), acetaminophen with codeine (moderate pain), morphine (severe pain).
I. Follow up.
1. Routine well-child checks:
a. Birth to 6 months: every 2 months, CBC every visit.
b. 6 months to 2 years: every 3 months.
• CBC every 3–6 months.
• Urinalysis (UA) annually.
• Ferritin or serum iron and total iron-binding capacity (TIBC) once at 1-2 years of age.
• BUN, creatinine, liver function tests (LFTs) once at 1-2 years of age.
• Influenza vaccine annually.
• Start folic acid daily at 1 year of age.
c. 2-5 years: visits every 6 months.
• CBC and UA at least yearly.
• BUN, creatinine, LFTs every 1-2 years.
• 23-valent pneumococcal vaccine at 2 years of age, with booster at 5 years.
• Hearing, vision, purified protein derivative (PPD) per standard practice.
d. Older than 5 years: visits every 6-12 months.
• CBC and UA at least annually.
• BUN, creatinine, LFTs every 2-3 years.
• Influenza vaccine yearly.
• Continue folic acid.
• May opt to stop penicillin V.
e. Adolescent: yearly visits.
• CBC and UA yearly.
• BUN, creatinine, LFTs every 2-3 years.
• Ferritin or serum iron and TIBC at least once.
• Hearing, vision, PPD as per standard practice.
• Influenza vaccine yearly.
2. Close monitoring by family for complications.
J. Complications.
| | |
| Acute chest syndrome, 517.3 | Priapism, 607.3 |
| Anemia, 285.9 | Renal dysfunction, 593.9 |
| Aplastic crisis, 284.9 | Renal failure, 586 |
| Avascular necrosis of hips, 733.42 | Retinal detachment, 361.9 |
| Chronic lung disease, 518.89 | Retinopathy, 362.1 |
| Febrile events, 780.6 | Splenic sequestration, 289.52 |
| Gallstones, 574.2 | Stroke, 436 |
| Hemolysis, 283.9 | Vaso-occlusive events, 459.9 |
| Leg ulcers, 707.1 | |
| |
1. Acute and chronic complications (hemolysis, anemia, gallstones) need early recognition, prompt treatment to reduce morbidity and mortality.
2. Acute events:
a. Painful vaso-occlusive events.
b. Febrile events.
c. Acute chest syndrome.
d. Splenic sequestration.
e. Stroke.
f. Aplastic crisis.
3. Chronic events:
a. Avascular necrosis of hips/shoulders.
b. Priapism.
c. Chronic lung disease.
d. Leg ulcers.
e. Renal dysfunction or renal failure.
f. Retinopathy and retinal detachment.
K. Education.
1. When family/patient should seek immediate medical care:
a. Fevers or persistent low-grade fever.
b. Pain unrelieved by prescribed oral medications.
c. Persistent abdominal pain, recurrent emesis.
d. Dyspnea, pain with breathing, hemoptysis, or feelings of tightness in chest.
e. Angina, exercise intolerance, or orthopnea.
f. Visual/speech changes, weakness/numbness in extremities, persistent headaches.
g. Sustained penile erections unrelieved by prescribed medications.
h. Increased lethargy, irritability, or pallor.
2. Factors that may precipitate painful vaso-occlusive crises:
a. Inadequate rest, emotional stress, fatigue.
b. Vasoconstrictive drugs, smoking, constrictive clothing.
c. Dehydration, strenuous physical exercise.
d. Extreme hot/cold temperatures, high altitudes, unpressurized aircraft.
V. BLEEDING DISORDERS
A. Usually bruising and bleeding are a part of active childhood. Bruises on the knees and shins and nose bleeds can simply be a fact of life. They become a health problem when the bleeding is hard to control or the bruising is in areas of the body that one does not normally hit, e.g., the back or stomach. Also, in adolescence, heavy menses could be the norm for many girls or it could signal a bleeding problem. The provider needs to decide when further studies would differentiate pathological or nonpathological causes of the bleeding and bruising. Normal clotting involves some 20 clotting factors that work together along with some other chemicals to form a substance called fibrin that leads to a clot.
B. Etiology.
1. Platelets or clotting factors do not work in the right way.
2. Supply of specified clotting factors are deficient.
3. Physical abuse.
4. Vitamin K deficiency.
C. Occurrence.
1. Congenital.
a. Hemophilia A or B–about 400 newborns a year are diagnosed in the U.S.
• X-linked recessive pattern of inheritance.
• Deficiencies in factor VIII (hemophilia A) and factor IX (hemophilia B).
• Males exclusively affected, some very rare cases of females with hemophilia.
b. von Willebrand–affects 66-100 people/1 million of the population.
• Autosomal inheritance with variable phenotypic expression.
• Common in both men and women.
• Deficiency in the von Willebrand clotting factor.
i. Blood protein that affects platelet function.
ii. Is a critical link between platelets and exposed vascular subendothelium.
iii. Binds and stabilizes coagulation factor VIII.
2. Acquired.
a. Acquired hemophilia.
• Rare condition with no genetic inheritance.
• Autoimmune etiology that results in development of autoantibodies to coagulation factors.
b. Disseminated intravascular coagulation.
• Acquired syndrome that activates the coagulation pathway.