Pediatric Thalassemia Treatment & Management

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

Surgical Care

Splenectomy is the principal surgical procedure used for many patients with thalassemia. The spleen is known to contain a large amount of the labile nontoxic iron (ie, storage function) derived from sequestration of the released iron. The spleen also increases RBC destruction and iron distribution (ie, scavenger function). These facts should always be considered before the decision is made to proceed with splenectomy. In addition, with recent reports of venous thromboembolic events (VTEs) after splenectomy, one should carefully consider the benefits and the risks before splenectomy is advocated. The spleen acts as a store for nontoxic iron, thereby protecting the rest of the body from this iron. Early removal of the spleen may be harmful (liver cirrhosis has occurred in such individuals).

In a retrospective study in which the charts and imaging studies of 89 patients with thalassemia intermedia were reviewed, renal stones were identified in 11 patients (12%) and 22 patients were on treatment for hyperuricemia (25%). The risk of renal stones seems to increase with age. Major identified risk factors for the formation of renal stones were splenectomy (91%) and higher number of erythroblasts. Such affected patients have higher mean creatinine levels and lower GFRs in comparison to nonaffected patients. [17]

Conversely, splenectomy is justified when the spleen becomes hyperactive, leading to excessive destruction of RBCs and thus increasing the need for frequent blood transfusions, resulting in more iron accumulation. Furthermore, if the labile iron pool in the spleen becomes the target for the action of the DFO (ie, removing the nonharmful pool and leaving the toxic one), splenectomy is further justified. The goal in this confusing dilemma should always be to achieve a negative iron balance, which, in many patients, has been possible by continuous administration of subcutaneous DFO.

Several criteria are used to aid in the decision for splenectomy; a practical one suggests that splenectomy may be beneficial in patients who require more than 200-250 mL/kg of PRBC per year to maintain an Hb level of 10 g/dL.

The risks associated with splenectomy are minimal, and many of the procedures are now performed by laparoscopy. Postsplenectomy risk of infections with encapsulated organisms and malaria in endemic areas is always a concern. The problem is minimal at the present time, since presplenectomy immunizations and postsurgical prophylactic antibiotics have significantly decreased the rates of such complications. Traditionally, the procedure is delayed whenever possible until the child is aged 4-5 years or older. Aggressive treatment with antibiotics should always be administered for any febrile illness while awaiting the results of cultures. Low-dose daily aspirin is also beneficial when the platelet count rises to more than 600,000/µL postsplenectomy.

Another surgical procedure in patients with severe thalassemia on transfusion therapy is the placement of a central line for the ease and convenience of administering blood transfusions, chelation therapy, or both.

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Consultations

The following consultations may be indicated:

  • Pediatric surgeon
  • Pediatric endocrinologist
  • Pediatric ophthalmologist
  • Pediatric otolaryngologist
  • Pediatric gastroenterologist
  • Pediatric HSCT specialist
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Diet

A normal diet is recommended, with emphasis on the following supplements: folic acid, small doses of ascorbic acid (vitamin C), and alpha-tocopherol (vitamin E). Iron should not be given, and foods rich in iron should be avoided. Drinking coffee or tea has been shown to help decrease absorption of iron in the gut.

In an animal study, green tea as antioxidant was shown to inhibit or delay the deposition of hepatic iron in thalassemic mice. [18] This prevented iron-induced free radical generation, which has been implicated in liver damage and fibrosis.

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Activity

Patients with well-controlled disease are usually fully active. Patients with anemia, heart failure, or massive hepatosplenomegaly are usually restricted according to their tolerances.

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