Pediatric Thalassemia Medication

Updated: Aug 23, 2017
  • Author: Hassan M Yaish, MD; Chief Editor: Max J Coppes, MD, PhD, MBA  more...
  • Print
Medication

Medication Summary

Medications needed for the treatment of various types of thalassemias are nonspecific and only supportive. A list of such medications is provided in this article.

Next:

Antipyretics, analgesics

Class Summary

Administration before blood transfusion prevents or decreases febrile reactions.

Acetaminophen (Tylenol, Tempra, Panadol)

Antipyretic effect through action on hypothalamic heat-regulating center. Action equal to that of aspirin but preferred because does not have adverse effects of aspirin.

Previous
Next:

Antihistamines

Class Summary

Administration prior to blood transfusion may decrease or prevent allergic reactions.

Diphenhydramine hydrochloride (Benadryl)

Antihistamine with anticholinergic and sedative effects.

Previous
Next:

Chelating agents

Class Summary

These agents are used to chelate excessive iron from the body in patients with iron overload.

Deferoxamine mesylate (Desferal)

Chelates iron from ferritin or hemosiderin but not from transferrin, cytochrome, or Hb.

Deferasirox (Exjade)

Deferasirox comes in tablet form for oral suspension. It is an oral iron chelation agent that reduces liver iron concentration and serum ferritin levels. Deferasirox binds to iron with a high affinity, in a 2:1 ratio. It is approved to treat chronic iron overload due to multiple blood transfusions and nontransfusion-dependent thalassemia.

Previous
Next:

Corticosteroids

Class Summary

Some patients may develop local reaction at the site of DFO injection. Hydrocortisone in the DFO solution may help to reduce the reaction.

Hydrocortisone (Solu-Cortef, Cortef, Hydrocortone)

Anti-inflammatory action. Both Na succinate (Solu-Cortef) and Na phosphate (Cortef) forms used for IV infusion, but not Na acetate form (Hydrocortone).

Previous
Next:

Antibacterial combinations

Class Summary

Certain antibacterial agents are known to be effective against organisms that often cause infection in patients with iron overload who also are receiving DFO therapy. Although rare in healthy patients, Y enterocolitica requires siderophores; thus, infections with this pathogen occur with relative frequency in patients with thalassemia. Appropriate therapy is a combination of trimethoprim-sulfamethoxazole (TMP/SMX) and gentamicin. Patients who require splenectomy need to receive prophylactic penicillin to prevent fulminating sepsis, especially those younger than 5 years. Many recommend that older patients receive prophylactic antibiotics for at least 3 years after splenectomy.

Trimethoprim-sulfamethoxazole (TMP/SMX, Bactrim, Septra)

In combination with gentamicin, DOC for infections by Y enterocolitica.

Gentamicin (Garamycin)

Aminoglycoside known to be effective against gram-negative microorganisms. Dosing regimens are numerous; adjust dose based on CrCl and changes in volume of distribution.

Penicillin V (Pen-Vee, Veetids, V-Cillin K)

DOC for postsplenectomy prophylaxis; erythromycin used in patients allergic to penicillin. Active against most microorganisms considered to be major offenders in splenectomized patients, namely, streptococcal, pneumococcal, and some staphylococcal microorganisms, but not penicillinase-producing species.

Previous
Next:

Vitamins

Class Summary

Several vitamins are required, as either supplements or enhancers of the chelating agent.

Serum level of vitamin C is low in patients with thalassemia major, likely due to increased consumption in the face of iron overload.

Ascorbic acid (Vitamin C, Cebid, Vita-C, Ce-Vi-Sol, Cecon, Dull-C)

Delays conversion of transferrin to hemosiderin, thus making iron more accessible to chelation.

Alpha-tocopherol (Vitamin E, Aquasol E, Vita-Plus E Softgels, Vitec, E-Vitamin)

An antioxidant. Prevents iron-mediated toxicity caused by peroxidation of cell membrane lipids, reducing extent of accompanying hemolysis. Protects polyunsaturated fatty acids in membranes from attack by free radicals and protects RBCs against hemolysis. Demonstrated to be deficient in patients with iron overload receiving chelation therapy.

Folic acid (Folvite)

Required for DNA synthesis; therefore in great demand in these patients because of increased cellular turnover. Deficient in most patients with chronic hemolysis.

Previous
Next:

Vaccines

Class Summary

Splenectomized patients are usually prone to developing infections with the encapsulated organisms such as pneumococci, Haemophilus influenzae, and meningococcal organisms. For this reason, such patients now are immunized against these organisms 1-2 wk prior to the procedure to prevent infections.

Pneumococcal vaccine polyvalent (Pneumovax)

Polyvalent polysaccharide vaccine (PS23) contains 23 serotypes that cause 70% of invasive infections. This vaccine should not be given to children < 2 y. In rare cases in which splenectomy is required in children < 2 y and no previous vaccination has been given, conjugate type (PCV7), which contains only 7 serotypes, is required.

Haemophilus influenza type b vaccine (ActHIB, HibTITER, PedvaxHIB)

Used for routine immunization of children against invasive diseases caused by H influenzae type b. Decreases nasopharyngeal colonization. The CDC's Advisory Committee on Immunization Practices (ACIP) recommends that all children receive one of the conjugate vaccines licensed for infant use beginning routinely at age 2 mo.

Conjugate form usually given in series of 3 doses at ages 2, 4, and 6 mo. Patients who have already received primary vaccine and booster dose at age 12 mo or older are usually protected and do not require further vaccination prior to splenectomy.

Meningitis group A C Y and W-135 vaccine (Menomune-A/C/Y/W-135)

Used only in children >2 y. Serogroup specific against groups A, C, Y, and W-135 Neisseria meningitidis.

Pneumococcal 7-valent conjugate vaccine (Prevnar)

Sterile solution of saccharides of capsular antigens of S pneumoniae serotypes 4, 6B, 9V, 14, 18C, 19F, and 23F individually conjugated to diphtheria CRM197 protein. These 7 serotypes have been responsible for >80% of invasive pneumococcal disease in children < 6 y in the United States. Also accounted for 74% of penicillin-nonsusceptible S pneumoniae (PNSP) and 100% of pneumococci with high-level penicillin resistance. Customary age for first dose is 2 mo but can be given to infants as young as 6 wk. Preferred sites of IM injection are anterolateral aspect of the thigh in infants or deltoid muscle of upper arm in toddlers and young children. Do not inject vaccine in gluteal area or areas that may contain a major nerve trunk or blood vessel. A 3-dose series, 0.5 mL each, is initiated in infants aged 7-11 mo (4 wk apart; third dose after first birthday).

Children aged 12-23 mo are given 2 doses (2 mo apart). Children >24 mo through 9 y are given 1 dose. Minor illnesses, such as a mild upper respiratory tract infection, with or without low-grade fever, are not generally considered contraindications.

Previous
Next:

Antineoplastic agent

Class Summary

Some patients may respond to hydroxyurea and subsequently decrease or eliminate transfusion requirements. Patients with homozygous or heterozygous XmnI polymorphism were found to respond favorably in one study. [19] Improvement of pulmonary hypertension following hydroxyurea has also been observed. [20]

Hydroxyurea (Droxia, Hydrea)

Inhibitor of deoxynucleotide synthesis.

Previous
Next:

Growth Hormone

Class Summary

Excessive chelation with deferoxamine may cause growth retardation. Growth hormone may be effective in increasing growth rate in all thalassemic patient particularly the ones with growth hormone deficiency. [21]

Somatropin (Saizen, Genotropin, Humatrope, Norditropin, Tev-Tropin)

Human growth hormone produced by recombinant DNA technology (mouse C127 cell line). Elicits anabolic and anticatabolic influence on various cells including: myocytes, hepatocytes, adipocytes, lymphocytes, and hematopoietic cells. Exerts activity on specific cell receptors including insulinlike growth factor-1 (IGF-1).

Previous