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Sunday, February 9, 2014

HEMOLYTIC ANEMIAS its classification,clinical features,diagnosis, and newest treatment

HEMOLYTIC  ANEMIAS


Definition:
A hemolytic anemia is a reduction in the number of circulating red cells resulting from their premature destruction.  
Pathophysiology:
        When the lifespan is shortened , the circulating red cell mass and supply of O2 is decreased  lead to increase erythropoietin and so increase red cell mass.
        When the rate of destruction exceeds 8 times normal so hemolytic anemia results.
Site of Destruction:
Intravascularly, extravascularly or more commonly in both sites .
Intravascular hemolysis :
When Hb  is released into the circulation it binds to haptoglobin .
          - The plasma haptoglobin level falls .
        .   - Haemoglobinuria and Haemosiderinuria

Compensatory Mechanism:
               *Erythroid hyperplasia.   
         *Change in the myeloid / erytheroid ratio       
        *Expansion of the marrow cavities of the skull,        vertebrae and long bones , causing marked                skeletal deformities.
    . Pressure effects on nearby organs     * 
Pathological changes in haemolytic anaemia:
increased red cell destruction Effects of         Elevated unconjugated bilirubin -
         - Reduced or absent haptoglobins
       .  - Elevated methaemalbumin
       . - Increased urinary urobilinogen.
          - Haemosiderinuria  ( chronic intravascular            haemolysis ).       
        - Reticuloendothelial hyperplasia – hepatomegaly and splenomegaly.
Effect of increased red cell production:
       .  Rise in reticulocyte count.
      .  Bone marrow expansion – hypercellularity .


Classification
      * Extrinsic factors ( e.g. antibody –complement mediated lysis or direct trauma).
      * Membrane abnormality ( e. g  congenital spherocytosis).
     * Abnormalities of red cell metabolism ( e.g glucose –6 phosphate dehydrogenase -G6PD – deficiency).
     * Disorders of haemoglobin ( e.g  thalassaemia or sickle    cell disease).

I –Red Cell Enzyme Deficiencies
 
The red cell has a very simple metabolism ; it need little more than  supply of sugar from which it obtains energy by two metabolic routes.
      1-The anaerobic Embden - Meyerhof                                                    pathway   
      2-Aerobic pentose phosphate pathway

Substances generated are:
        *ATP , drive the anion membrane pumps which         remove sodium and water from cell .       
 * 2-3 diphosphoglycerate alters the oxygen affinity        of hemoglobin .    
     * NADH , maintaining the iron atom of                          hemoglobin in the reduced (Fe) state.
      * NADPH , important for maintaining sulphydry groups (-SH) in the reduced state .         
Glucose –6 Phosphate Dehydrogenase Deficiency
       . X- linked trait -
       . - The disorder is extremely heterogenous
       - Affected Negroes have 10 – 15 % normal enzyme activity in their red cells.
       - There are two – types of G6PD – A and B 
 Type A Negro population.            
Type B all population .             
The gene for G6PD is located on the long arm of the X- chromosome.
Affected males have very low or absent activity in their red cells.Clinical features
G6PD deficiency may cause
      . Drug induced hemolytic anemia.     
       . Favism.     
       . Neonatal jaundice .     
     . Chronic hemolysis  .

Oxidant drugs
The most important offenders are the sulphonamides  and the anti malarials primaquine and chlorquine.
  
Ingestion of the drug is followed rapidly by intravascular haemolysis with fever , malaise , prostration and passage of dark urine.
  
The haemoglobin (Hb) level falls rapidly , accompanied by a marked reticulocytosis and the appearance of Heinz bodies in many of the red cells.

 
Favism: 
        Acute haemolytic episodes following ingestion of        the fava beans.  
Neonatal jaundice:
            - Occur up to the third week of life.
       .     - Leads to a full picture of kernicterus
Chronic haemolysis:
      .       - A few reported families
             - The hemolysis in such circumstances may         be exacerbated by drugs or intercurrent infection.
          -Splenectomy is of only limited value.   
Diagnosis: 
A simple assay for G6PD activity.
Pyruvate Kinase Deficiency  
         * Is the commonest enzyme  deficiency in the Embden – Meyerhof pathway.
         * Autosomal  recessive disorder.
         * Subnormal levels are observed in the parents of affected children.  
Clinical feature:
     .  Presents in childhood   -
         - Anemia , jaundice and splenomegaly , or sometimes even earlier as neonatal jaundice.   
         - The clinical course is characterized by variable degree of anemia exacerbated by infection or other stress.
         - There is an increased incidence of gallstones and bone changes.
Pyruvate Kinase Deficiency  
         * Is the commonest enzyme  deficiency in the Embden – Meyerhof pathway.
         * Autosomal  recessive disorder.
         * Subnormal levels are observed in the parents of affected children.  
Clinical feature:
     .  Presents in childhood   -
         - Anemia , jaundice and splenomegaly , or sometimes even earlier as neonatal jaundice.   
         - The clinical course is characterized by variable degree of anemia exacerbated by infection or other stress.
         - There is an increased incidence of gallstones and bone changes.
Investigations :  
Low or absent  PK activity in red cells .  
Treatment : 
        Splenectomy .   
             . Hemolytic crisis are treated by            blood  transfusion .         

II – Haemoglobinopathies
        - Inherited disorders of hemoglobin structure or its production .      
        - Human hemoglobin in a globular protein , it          consists of    a protein part , globin , combined          with four hem groups .
         -  The globin fraction of normal adult                        hemoglobin   (Hb A ) consists of four peptide          chains , two (a) - chains  and two (b) chains.
        
    - Fetal hemoglobin (Hb F )  has two (a) chains and  two             (g) chains.     
          -  Normal adults have a minor hemoglobin (Hb A 2 ) with (a) chains and (d) chains .       
 
  - Inherited defect in the rate of synthesis of one or  more of         the globin chains results in under  production of                   hemoglobin in unbalanced  globin chain synthesis with        perception of the chains which are produced in                              excess , and the formation of rigid inclusion
                ( Heinz bodies ) .               
     (A)   Structural Haemoglobinopathies
Three main types
       Hemoglobin variants which cause hemolytic alteration of the molecule configuration of Hb (sickling) of Heinz body formation .
Abnormal hemoglobin with altered oxygen carrying properties hereditary methemoglobinaemia or polycythemia.
     Variant which are synthesized at a reduced rate, (thalassaemia ).
(A) Sickling disorders:
        During deoxygenation the abnormal hemoglobin molecule form linear stricks , causing the red cell to become deformed into a rigid , sickle shape .
·        Sickle cells have difficulty in passing through the microcirculation , this leads to blockage of small vessels and tissue damage .
        Sickling damage the red cell membrane and leads to haemolysis .

Sickle cell anaemia (ss)
·         - Severe , in capacitating , disorder resulting in hemolytic anemia from about the third month of life .
 - Chronic hemolytic anemia punctuated by periodic exacerbations or crisis .

Diagnosis of sickling disorders :
            1-The racial background with typical                     clinical  and hematological feature   
          2-Hematological findings       
           3- A sickling test
         4- Screening tests
      5- The diagnosis should always be confirmed by            hemoglobin electrophoresis       
Sickle cell disease crisis
   1- Painful crises
          - Results of either diminished  blood flow leading to               tissue infarction or sequestration of sickled                                                                                                                                             erythrocytes  in various organs .        
          - May be precipitated by factors including infection ,              dehydration and exposure to low temperatures .   
·           - In the first year of life a common presenting feature is        the painful swelling of the hands and feet (hand –foot-          syndrome) .    
         - In older children and adults , sever pain  in the limbs             and  back associated with fever and  prostration is the                    commonest presentation .

- 2-Aplastic crises  
         Rapidly falling hemoglobin level together          with   an absent reticulocyte count .    

- 3-Sequestration crisis  
                Pooling of sickled erythrocytes may occur in            the  spleen liver or lungs .    
               Life –threatening complication seen in early              childhood .   
      Characterized  by massive enlargement of the spleen      together with a sharply falling hemoglobin level        and rising of the reticulocyte count
        Splenic infarction occurs between 5 and 10 years of age so this complication is not seen in adults .

        Occur in the liver in adult life , leading to rapid enlargement together with a falling hemoglobin level and a rise in reticulocyte count .
       Occur in the lungs where bilateral radiological changes associated with tachycardia , fever and profound hypoxia may accompany a rapidly falling hemoglobin level .
 Other complications  
 Infection
Pneumococcal infections , and prophylactic penicillin together with vaccination should be considered . Salmonella osteomyelitis .
Renal damage
   o       Impairment of tubular function
   o       Infarction leading to hematuria    
   o       Papillary necrosis and
o       Nephrotic syndrome           
Chronic leg ulceration.
Aseptic bone necrosis
Priapism  .
Gallstones
Pulmonay  
Management : Between crises

  o  Folic acid
  o  Any infections treated promptly    
  o   Painful crises should be treated with rest ,                         hydration , and adequate analgesia .
  o   Hospitalization may be required for sever crises
  o   Hemoglobin level and reticulocyte count must               be performed at least daily during severe                     crises   
      Routine blood transfusion is unnecessary .
o  Transfusion may be indicated , if the hemoglobin falls much below its usual level.
o  Vaso occlusive complications of sickling are prevented if the proportion of HbS – containing cells is reduced to less than 30% .
o Sodium cyanate increase O2 affinity of HBS but it’s long tern use results in neurotoxicity , extra – corporeal treatment of the sickeled red blood cells with cyanate is now available and useful. . Prevention
      1-Genetic counseling
    Pre- natal diagnosis and termination of pregnancy 2- 
(B) The B- thalassaemias
Thalassaemia Major , Cooley’s Anaemia  
        Sever form .
      Manifest in infancy .
       Prognosis is grave .
       Many fail to survive into adult life .
       The children stunted .
       Anemia is usually severe .
        Jaundice is mild.
       Splenomegaly is often .
 . Enlargement of the liver
       . Erythrocytes very deficient in hemoglobin with great variation in shape and size , target cells are quite common and basophilic stippling.
       Leucocytosis .
        Platelet count is normal .
        The reticulocytes count is raised .
      .The bone marrow usually shows a normoblastic hyperplasia expand the marrow cavities with great widening of the diploe and very prominent vertical striations are to be seen on X- ray as hair on end and mongoloid appearance.
       The spleen usually is greatly enlarged .



Thalassaemia minor and Minima
       Benign condition -
       - Detected incidentally in apparently normal individuals mild anaemia.
      - Most of these patients are completely symptoms – free.
     The haemoglobin  level may be in the normal range.    - The blood film appearances are similar   in type to those of thalassaemia major but are much less marked .   
      .%  - The reticulocyte count is rarely over 5
     .  Only small amounts of haemoglobin F are found
      .  High levels of haemoglobin A2 -

thalassaemias a
Haemoglobin Barts – Hydrops Fetalis Syndrome:  
     .   - This condition is incompatible with life
          - The only chains synthesized are g and b       chains.      
      . -The clinical picture is of hydrops fetalis
     - Haematological picture is of severe thalassaemic syndrome.  




Haemoglobin H –Disease:
        Is a B –chain tetramer .
         . The B- chian tetramer is unstable and becomes                 precipitated within the erythrocytes
       These cause membrane damage .
        . Are important cause of the premature erythrocyte             destruction . 
     Enhanced by oxidant drugs .
       The results of splenectomy are difficult to assess .
        Many avoid the use of any oxidant drugs .

Management of the Thalassaemias  
In the very mild or silent forms of thalassaemia no treatment is usually indicated.

        Folate supplements are therefore usually given.
      General medical care.
        Prompt treatment for any episode of infection.
 In the more severe forms of homozygous B- thalassaemia , periodic blood transfusion is the only way of maintaining a reasonable haemoglobin concentration   ®hyper transfusion .
                          super transfusion  

        Splenectomy is usually only contemplated in children with severe B- thalassaemia , indication :

evidence of excessive erythrocyte destruction .
sequestration .
        Population screening and genetic counseling .
Problem of iron overload :
Excess iron deposition in all organs which result in tissue hemosiderosis (2ry hemochromatosis ), with the clinical picture of :
-1-Hepatic dysfunction
-2-Growith failure
-3-Cardiac complications
Treatment of iron overload :
Chelation therapy by desferroxamine (Desferal ) given by small mechanical infusion pump of 1.5 gm over a period of 8-12 hours ,  3- 5 times /week.
      


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