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Management of transfusion dependant thalassemia.

Transfusion dependant thalassemias:

  • 80-85% of homozygous β° thalassemias
  • 25% of homozygous β+ thalassemias

Treatment:

1. Guidelines for Transfusion therapy:

  • RBC depleted of leukocytes
  • Matched for D,C,c,E,e & kell Ag
  • Cytomegalovirus safe units for stem cell transplantation candidates
  • Usual intervals 3-4wks
  • Hb goal 9.5-10.5g/dl
    • Monitor for
    • Transfusion ass infections
    • Transfusion ass reactions
    • Alloimmunization

2. Monitoring for iron overload:

Serum ferritin: screening technique
R2 MRI Quantitative liver Iron: best indicator usually started at 10yr.
Chelation therapy:
Chelation used in case of iron overload
General after 1 yr of transfusion
S. Ferritin > 1000ng/ml
Liver Iron >5000μg/g dry weight
Not <2yrs of age
Goals
  1. Prevent hemosiderosis induced tissue injury
  2. Avoid chelation toxicity.
Chelators

Deferoxamine

Deferasirox

Deferiprone

I.V / S.C

Oral

Oral 2nd line

8hr/d for 5-7days/wk

O.D

T.D.S

25mg/kg - 60mg/kg

20mg/kg/d -

40mg/kg/d

75mg/kg/d -

99mg/kg/d

Excellent safety

and efficacy

Adherence

Easily enter cardiac & other tissues

Skin reaction

Ototoxic

Retinal changes

Bone dysplasia

Truncal shortening

Kidney damage

G.I symptoms

Hepatic transaminitis

Transient Agranulocytosis

C.I when febrile


3. Hydroxyurea:

Used in β thalassemia intermedia
Dose: 10mg/kg - 20mg/kg
Mean increase in Hb is 1gm/dl
Reduced risk of
    • Leg ulcers
    • Pulmonary HTN
    • Extramedullary hematopoiesis
Risk of developing cytopenia

4. Hemopoietic stem cell transplantation:

In low risk HLA matched siblings
- survival rate is 90%
- event free survival is 80%
Gene therapy with lentiviral vectors for no donar pts 

5. Splenectomy:

Indications:
  • hypesplenism
  • falling steady state Hb
  • ↑ transfusions frequency
Adverse effects:
  • Infections risk
  • Venous thrombosis
  • Pulmonary HTN
  • Leg ulcers
  • Silent cerebral infarction
Prophylaxis:
  • Prophylactic penicillin
  • Immunization against capsulated bacteria

Preventive monitoring of Thalassemia pts:

1. Cardiac disease:

Major cause of death
Periodic electrocardiogram from 10yrs of age - risk of arrhythmias
Serial electrocardiogram to monitor
  • Cardiac function
  • Pulmonary artery pressure (Pulmonary HTN frequently occurs in non transfusion dependant pts - indication of BT)
After 8yrs chronic transfusions - cardiac hemosiderosis may occur
  • Require T2 MRI for diagnosis
  • Intense combination chelation therapy

2. Endocrine disease:

↓ endocrinal functions usually after 5yrs age/ 3yrs transfusion therapy due to
  • Hemosiderosis in pituitary and other glands causes
    • Hypothyroidism
    • G.H deficiency
    • Hypo parathyroidism
    • Diabetes mellitus
    • Osteoporosis
    • Adrenal insufficiency
  • Nutritional deficiencies
Measures:
Bi-annual: Height, weight, pubertal development
From 10yrs age: Bone scan
Vit D, Vit C, Zn supplementation

3. Psychological support:

  • Culturally sensitive anticipatory counseling
  • Early use of child life services
  • Early social service consultation for financial & social issues

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