Bone Marrow Transplantation,Neocyte Transfusion,Splenectomy:Thalassemia Management

4th Pediatric Infectious Diseases Conference

ISSN 0973 - 0958

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Hemoglobin Variants Detection by High Performance Liquid Chromatography Method

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Results

MODERN TRENDS IN MANAGEMENT OF THALASSEMIA

Related Topics

Thalassemia Epidemiology and Pathophysiology

b-thalassemia Clinical Manifestations,Diagnosis and Management

Transfusion Therapy in Thalassemia

Iron Overload and Chelation Therapy

Pharmacological Methods To Increase Gamma Chain Production and Gene Manipulation

Dr.M.R.Lokeshwar
Consultant Pediatric Hematologist,
Imperial Mahal, 3rd floor, Dadar TT, Mumbai - 400014.

SPLENECTOMY :

With the advent of hyper and super-transfusion therapy, splenomegaly and hypersplenism have become a rarity and hence splenectomyis usually not needed in these patients.
If the child has already developed splenomegaly and signs of hypersplenism and is above 5 years of age, splenectomy is indicated.
The indications for splenectomy are :
- An increase in the yearly requirement of packed cells more than double the basal requirement i.e. packed cell 200 cc/kg/year or more.
- Decrease in WBC and platelet count, which is a late manifestation of hypersplenism.
All children needing splenectomy should receive pneumococcal vaccine (pneumovax MSD), H-influenza vaccine, and meningococcal vaccine 4 weeks prior to surgery. In endemic areas, prophylactic antimalarial treatment may be given to prevent malaria.
Prophylactic penicillin therapy must be continued life-long.
Episodes of infection should be treated promptly and newer antibiotics may be empirically started to prevent septicemia and other complications (if necessary these children should be hospitalized). Blood culture and sensitivity of antibiotics must be performed to guide treatment.

BONE MARROW TRANSPLANTATION :

A ray of hope for permanent cure and better future for children with genetic disorders has brightened with the rapid advancement in the techniques and the success of bone marrow transplantation.

The credit of first bone marrow transplantation in thalassemia major goes to E.Donald Thomas who performed this procedure in an 18 month old thalassemic child in 1982 using HLA matched elder sister as donor. This child was cured of thalassemia. The first BMT in thalassemia in India was successfully done by Dr.M.Chandi at Christian Medical Collage, Vellore.
The principles of bone marrow transplantation in thalassemia are:
- To destroy and prevent regeneration of defective stem cells,
- sufficient immune suppression for good engraftment of normal marrow,
- to infuse stem cells with normal gene for b globin.
- To prevent GVHD with high doses therapy of Busulphan, Cyclophosphamide, total body irradiation and other modalities.
  
  All over the world, over 1000 transplantations have been done with a 70-80% cure rate.
  - The three most important adverse prognostic factors for survival and event-free survival are the presence of hepatomegaly (liver more than 2 cms. below costal margin), portal fibrosis and iron overload. (Lucarelli et al).
  - Bone marrow transplantation is most successful in patients who are young, properly transfused, well-chelated and in good clinical shape without hepatomegaly.
  - The cost of BMT in India is around Rs.4-5 lacs and is being done at Christian Medical Collage, Vellore and Tata Memorial Hospital,Parel, Mumbai, and AIIMS in Delhi.

NEOCYTE TRANSFUSION :

Propper et al in 1980, introduced the concept of transfusing thalassemic children with young red cells (Neocytes).
In conventionally used unit of blood, red cells have a survival of 60 days. The mean age of a neocytes being 120 days, they survive in the recipient for 90 days, thus reducing the amount of blood required and prolonging the interval between two transfusions.
IBM-2991 can be utilized for both washing the red cells and dividing them into populations of different ages by differential centrifugation technique.
There is a reduction in the total units of blood transfused and iron overload.
However this is not cost effective. The requirement of blood donors is increased and it requires costly equipment, technology and highly developed transfusion services. It is also time consuming.