This note will help you in knowing about childhood anemia's like iron deficiency, SCD etc.. also some of the bleeding disorders are also explained in this.

1. ANEMIAS
Mrs. Babitha K Devu
Assistant Professor
SMVDCoN

2. ANEMIAS

3. ANEMIAS
Anaemia is a condition in which the number
of red blood cells (and consequently their
oxygen-carrying capacity) is insufficient to
meet the body’s physiologic needs.
Specific physiologic needs vary with a
person’s age, gender, residential elevation
above sea level (altitude), smoking
behaviour, and different stages of
pregnancy.

4. ANEMIAS
Iron deficiency is thought to be the most common
cause of anaemia globally, but other nutritional
deficiencies (including folate, vitamin B12 and
vitamin A), acute and chronic inflammation,
parasitic infections, and inherited or acquired
disorders that affect haemoglobin synthesis, red
blood cell production or red blood cell survival,
can all cause anaemia.
Haemoglobin concentration alone cannot be used
to diagnose iron deficiency.

5. ANEMIAS
DEFINITION:-
Anemia is the reduction in the number &
quality of circulating RBC when the Hb level
is below the normal that required for a
particular age resulting in decreased oxygen
carrying capacity.

6. ANEMIAS
Hb level for different age groups:-
Population Non-
Anemia
Anemias
Mild Moderate severe
Children 6 - 59
months of age
110 or
higher
100-
109
70-99 < 70
Children 5 - 11
years of age
115 or
higher
110-
114
80-109 < 80
Children 12 - 14
years of age
120 or
higher
110-
119
80-109 < 80

7. ANEMIAS
Hb level for different age groups:-
Population Non-
Anemia
Anemias
Mild Moderate severe
Non-pregnant
women (15 years of
age and above)
120 or
higher
110-
119
80-109 < 80
Pregnant women 110 or
higher
100-
109
70-99 < 70
Men (15 years of
age and above)
130 or
higher
110-
129
80-109 < 80

8. ANEMIAS
Grading of Anemia (WHO/ICMR)

9. ANEMIAS
Clinical Grading of Anemia
Clinical Observation Grades
•Pallor restricting itself to
conjunctive and/or mucus
membrane only
Mild
•Pallor observed in skin and
appendages
Moderate
•Pallor observed in palmar
creases
Severe

10. ANEMIAS
Classification According to RBC
Morphology

11. ANEMIAS
Classification According to RBC Morphology
• Variaton in Size
Microcytosis: RBCs smaller than the normal size
are considered as microcytes. It is seen in : Iron
deficiency anemia, thalassemia, lead poisoning,
sideroblastic anemia and anemia of chronic
disorders.
Macrocytosis: RBCs larger than the normal size. It
is seen in : Liver diseases, hypothyroidism,
megaloblastic anemia, chemotherapy, post
splenectomy etc.

12. ANEMIAS
Classification According to RBC Morphology
• Variaton in color
RBCs that appear disc shaped and having an area of
central pallor that occupies approximately one-third of
the cell’s diameter (containing normal amount of
hemoglobin) are considered as normochromic RBCs.
Hypochromic : RBCs that have an area of pallor that is
larger than the normal are called hypochromic. seen in :
Iron deficiency anemia, anemia of chronic diseases,
thalassemia, some hemoglobinopathies, sideroblastic
anemia and any of the conditions leading to
microcytosis.

13. ANEMIAS
Classification According to RBC
Morphology
Microcytic hypochromic
Anaemia
RBC is small and less
colored
Normocytic
normochromic Anaemia
Found in impaired cell
production & haemolysis.
RBC appears normal
Macrocytic Anaemia RBC appears large

14. ANEMIAS
Causes of Anemia/Classification
Impaired Production of RBC
Deficiency Anemia (Fe, FA, Vit B12)
Bone marrow failure (Aplastic)
Impaired erythropoietin production
Miscellaneous (Congenital dyserythropoietic)

15. ANEMIAS
Causes of Anemia
Increased destruction of RBC (Hemolytic)
Hemolysis due to intrinsic factors – defects of
hemoglobin synthesis
Hemolysis due to extrinsic factors – are acquired
defects
Increased blood loss (Haemorrhagic anemia)
Acute blood loss
Chronic blood loss

16. ANEMIAS
Clinical Features
The early S/S might be following but it may
be asymptomatic in mild cases.
Fatigue
Listlessness/lassitude
Anorexia

17. ANEMIAS
Clinical Features
The late s/s are
Pallor (skin, nail bed, mucous membrane)
Easy fatigue and loss of energy.
Dyspnea, palpitation, headache on exertion
Vertigo, tinnitus, dizziness
Hepatomegaly & enalrgement of lymph gland
Hemic murmur becomes more prominent
depending on the severity increases.

18. ANEMIAS
Complications
Circulatory collapse & shock
CCF
Systemic/local infection
Growth retardation
Mental retardation
Delayed puberty

19. ANEMIAS
Diagnostic Study
Blood examination & counts
Bone marrow study
Serum Fe, Pb
Peripheral blood smear

20. ANEMIAS
Management
Depends upon the causes
Due to blood loss – control bleeding & restore
blood
Due to increased RBC destruction – treat specific
disorder
Due to decreased RBC production – control & treat
deficiencies

21. Iron Deficiency Anaemia/
IDA
• Every red blood cell in the body contains iron in its
haemoglobin, the protein that carries oxygen to the
body's tissues from the lungs. Iron gives
haemoglobin the strength to "carry" (bind
to) oxygen in the blood, so that oxygen gets to
where it needs to go.
• Iron helps move oxygen from the lungs to the rest
of the body and helps muscles store and use
oxygen. If your child's diet lacks iron, he or she
might develop a condition called iron deficiency.

22. Iron Deficiency Anaemia/
IDA
Definition:
It is common in infancy & is occurring due to
decreased RBC production due to the deficiency of
iron store resulting in microcytic hypochromic
anaemia.

23. Iron Deficiency Anaemia/
IDA
How much iron do children need?
Age group Recommended amount of iron a day
7 - 12 months 11 mg
1 - 3 years 7 mg
4 - 8 years 10 mg
9 - 13 years 8 mg
14 - 18 years, girls 15 mg
14 - 18 years, boys 11 mg

24. Iron Deficiency Anaemia/
IDA
Causes:
IDA is common in infancy because both breast as
well as cow milk do not provide the baby’s need’s
for iron.
Prematurity
Inadequate Fe storage in IU period
Twin pregnancy
Children who have certain health conditions, such
as chronic infections or restricted diets, infestation
Children ages 1 to 5 who have been exposed to
lead

25. Iron Deficiency Anaemia/
IDA
Causes:
Delayed weaning
Poverty
Increased need of Fe for preschooler and
adolescent due to rapid somatic growth
In older children. Inadequate intake, malabsorption,
infection, chronic blood loss
Adolescent girls also are at higher risk of iron
deficiency because their bodies lose iron during
menstruation.

26. Iron Deficiency Anaemia/
IDA
Pathophysiology:
Stage - 3
Fe deficiency
anaemia
In all lab test, Fe
status is abnormal
Microcytic
Hypochromic
Stage - 2
Iron deficient erythropoiesis
Stage - 1
Iron
Depletion
Iron storage is exhausted –
indicated by decreased Sr. Fe
level
No anaemia.
RBC morphology
is normal

27. Iron Deficiency Anaemia/
IDA
Pathophysiology:
Diminished Fe absorption in proximal small
intestine/intake or excessive loss of body Fe
Iron deficiency anemia
This can affect O2 transport, DNA synthesis &
electron transport. In severe, IDA the iron
containing enzymes are decreased which in turn
can affect immune & tissue function
Diminished growth & learning

28. Iron Deficiency Anaemia/
IDA
C/F:
Progressive pallor
Irritability
Anorexia
Tiredness and weakness
Cramps
Breathlessness
Tachycardia
FTT
Atrophy of tongue papillae

29. Iron Deficiency Anaemia/
IDA
C/F:
Glossitis, angular stomatitis, cheilosis
Pica
Koilonychias/platonychia
Pallor
Diarrhea
Spleenomegaly
Hemic murmur

30. Iron Deficiency Anaemia/
IDA
D/S:
Peripheral smear examination
MCV, MCH, MCHC
RBC distribution width (RDW)
Total RBC count
Sr. Fe and ferritin
TIBC
Transferrin saturation reduced

31. Iron Deficiency Anaemia/
IDA
Management:
The cause of anaemia should be identified and
corrected.
Hookworm infestation is the most common cause
of occult GI blood loss in rural India at all ages.
Dietary counselling & treatment of causative factors
are required to prevent recurrence or failure of
therapy.
Close follow up to assess for adequate response.

32. Iron Deficiency Anaemia/
IDA
Management:
Iron supplements
Oral therapy:
3-6mg/kg/day of elemental iron. Ferrous
sulphate (300mg in divided dose) is the most
effective & economical oral preparation. Best
absorbed when taken on an empty stomach or
in between meals. Ferrous gluconate and
ferrous fumerate also are good source.
Entric coated decreases side effects but less
efficacious & costly

33. Iron Deficiency Anaemia/
IDA
Management:
Iron supplements
Parenteral therapy:
The indications are:
• Intolerance to oral iron
• Malabsorption
• Ongoing blood loss at a rate where oral
replacement cannot match iron loss.

34. Iron Deficiency Anaemia/
IDA
Management:
Iron supplements
Parenteral therapy:
IV is preferred over IM route
IV iron sucrose is safe & effective
Dose 1-3mg/kg, diluted in 150ml of NS &
given as slow infusion over 30-90 minutes.
The total dose of parenteral iron can be
calculated by the following formula:

35. Iron Deficiency Anaemia/
IDA
Management:
Iron supplements
Parenteral therapy:
Iron required (mg) = wt (kg) x 2.3 x (15 – patient
Hb in g/dl) + (500 to 1000 mg)
The total calculated dose is given as divided
dose.
Iron dextran, ferric gluconate, ferric
carboxymaltose are some other drug of choice.
(IM should not be >5mg/kg in Z fashion )

36. Iron Deficiency Anaemia/
IDA
Management:
Blood Transfusion:
Blood transfusion should be avoided in,
stable patients
In severely anaemic child (Hb <4g/dl) BT
should be given only 2-3 ml/kg of packed
cells at any one time
Packed cell/ sedimented red cells should
be administered slowly.
Administer diuretics also if necessary.

37. Iron Deficiency Anaemia/
IDAManagement:
Dietary management:

38. Iron Deficiency Anaemia/
IDA
Management:
Dietary management:
Animal source are better absorbed than plant
source.
Vitamin C rich food should be also provided for
enhancing your body's absorption of iron
Calcium taken along with iron supplements will
inhibit the iron absorption.

39. Iron Deficiency Anaemia/
IDA
Prevention:
Adequate ANC
Prevention of pre-term delivery or LBW, &
control of infections in prenatal, natal, &
neonatal period.
Proper starting of weaning
Immunization
FE-FA supplementation to the children &
adolescent girls.
De-worming
Environmental sanitation

40. Iron Deficiency Anaemia/
IDA
Nursing Management:
Rest
Monitoring child condition
Emotional support
Maintaining hygienic care
Health education

41. Thalassemia
• Thalassemia is an inherited (i.e., passed from
parents to children through genes) blood
disorder caused when the body doesn’t make
enough of a protein called hemoglobin.

42. Thalassemia
• It is otherwise called Cooley Anaemia, or
Mediterranean Anaemia.
• The word is a Greek term derived from
“thalassa” which means ‘the sea’ & emia, which
means ‘related to blood’
• Prevalence: more common in populations in the
geographical belt from southeast Asia to Africa
• The carrier rates for beta thalassemia in north
Indians are reported to vary from 3-17% in
different ethnic groups.

43. Thalassemia
• Definition
• It is a group of hereditary haemolytic anaemia
characterized by reduction in the synthesis of
haemoglobin. It produces hypochromic
microcytic anaemia due to defective
hemoglobinization of RBC, haemolysis &
ineffective erythropoiesis.

44. Thalassemia
• Definition
• It is a chronic haemolytic anaemia, is an
inherited disorder of adult haemoglobin
synthesis which manifests as anaemia with
haemolytic faces, hepatosplenomegaly and
skeletal changes.
• It produces hypochromic microcytic anemia.

45. Thalassemia
• Etio-pathogenesis
Hereditary inability to synthesis globin
polypeptide chain
Due to gene
deletion/deficiency
α - chain synthesis
Point mutation/Single
base substitution
β - chain synthesis

46. Thalassemia
• Etio-pathogenesis
Ineffective erythropoiesis
Haemolysis due to immature, thin RBC with
short lifespan
Resulting in the formation of clinical
manifestations

47. Thalassemia
• Classification:
• Based on cause
–Alpha Thalassemia
–Beta Thalassemia
• Based on spectrum of Disease
–Thalassemia Major
–Thalassemia intermedia
–Thalassemia minor

48. Thalassemia
• Classification:
–Thalassemia Major: It is a severe form
of the illness & associated with
homozygous state. In this condition,
beta chain synthesis is markedly
reduced.
–Thalassemia intermedia: It is a state of
chronic hemolytic anaemia caused by
deficient α & β chain synthesis. It is also
a homozygous form.

49. Thalassemia
• Classification:
–Thalassemia minor: It is a mild form
of the illness produced by
heterozygosity of either alpha or beta
chain. It may be completely
asymptomatic or may have mild
anaemia, jaundice & abdominal pain.

50. Thalassemia
• Clinical Manifestations
• Manifested at the age of 3 months
• Progressive pallor
• Jaundice
• Hepato-spleenomegaly
• Recurrent respiratory infection
• Lymph node enlargement
• Growth failure

51. Thalassemia
• Clinical Manifestations
• In severe cases
– Mongoloid facial appearance:
bossing of the skull, prominent
frontal & parietal eminences
with flat vault & straight
forehead. Maxilla becomes
prominent with malformed
exposed teeth.
– Nose bridge depressed with
puffy eyes.

52. Thalassemia
• Clinical Manifestations
• In severe cases
– Anorexia
– Poor feeding
– Abdominal distension
– Irregular fever
– Increased pigmentation of the skin
– Hypogonadism
– Poor nutritional status with reduced activity level
– Skeletal changes & pathological fracture

53. Thalassemia
• D/S
• Blood examination
– CBC & peripheral blood film
• Bone marrow study
• Osmotic fragility test
• Radiological findings

54. Thalassemia
• Complications
• CCF, Hepatic failure
• Inter-current infection
• Gall stone
• Growth retardation
• Delayed puberty
• Hemosiderosis
• Transfusion related infections
• Endocrinopathies
• Skeletal complications
• Multi-organ dysfunction

55. Thalassemia
• Management
• Hematopoietic stem
cell transplantation:
it offers possibility
of cure in even
sever form. It is
expensive and is
available only to a
relatively small
number of patients.

56. Thalassemia
• Management
• Blood transfusion: regular
interval transfusion is
required to maintain the
Hb level at least 10 to 11
gm/dl. Usually 10-15ml/kg
every 2 to 3 weeks
washed packed RBCs are
transfused.

57. Thalassemia
• Management
• Iron Chelating Therapy: This is indicated
usually after 1-2 years of transfusion when
ferritin level is about 1000 – 1500 μg/l.
• The recommended drug is
Deferoxamine (DFOA), sold under the
brand name Desferal which must be
administered parentally because of its short
half –life.
• Prolonged SC infusion is the most effective
route.

58. Thalassemia
• Management
• Iron Chelating Therapy:
• A total dose of 40 – 60 mg/kg/day over 8-12
hours during night for 5-6 days a week by
mechanical pump.
• Warnings: Orange discoloration of urine
• If Cardiac failure occurs due to Fe overload,
then 6-10 g, IV deferoxamine is
administered.
• Eye, Ear & renal function test are required
to monitor the effect of therapy.

59. Thalassemia
• Management
• Iron Chelating Therapy:
• Other drugs with less effectiveness are:
– Deferiprone – Oral, 75mg/day
– Deferasirox – Oral, 30mg/kg/day
• Medication to prevent stem cell & blood
transfusion reactions:
– Acetaminophen & diphenhydramine HCl before
transfusion to reduce allergic reactions
– Hepatitis B vaccination & assessment of
hepatitis & HIV status

60. Thalassemia
• Management
• Splenectomy: To manage hypersplenism.
– Indication: Patient who require > 200-250 ml/kg
of packed RBC per year to maintain Hb.
Procedure is usually delayed until the child is
aged 7 year older.
• Supplementation:
– Calcium
– Vitamin D & bisphosphonates to improve bone
density
– FA

61. Thalassemia
• Management
• New Approaches: Human recombinant
erythropoietin (r-epo) – increases the level of Hb
in beta thalassemia intermedia thereby it will
reduce the need of blood transfusion.
• Chemotherapeutic agents: to stimulate
gammaglobulin chain synthesis
– 5-azacytidine- are used in the treatment of
myelodysplastic syndrome
– Hydroxyurea: increase HbF production
– Myleran: Used in some conditioning regimens
prior to bone marrow transplant.

62. Sickle Cell Anemia
• Sickle cell anaemia is an inherited form
of anaemia — a condition in which there
aren't enough healthy red blood cells to
carry adequate oxygen throughout your
body. In sickle cell anaemia, the red
blood cells become rigid and sticky and
are shaped like sickles or crescent moons.

63. Sickle Cell Anemia

64. Sickle Cell Anemia
• Definition:
Sickle cell anaemia is an autosomal
recessive disease that results from the
substitution of valine for gultamic acid at
position 6 of the beta-globin gene which
result in the formation of abnormal Hb
(HbS), causes chronic hemolytic anaemia.

65. Sickle Cell Anemia

66. Sickle Cell Anemia
• Who is at risk for sickle cell anemia?
• People from regions that have endemic
malaria are more likely to be carriers. This
includes people from:
• Africa
• India: gene frequency is 4.3%
• the Mediterranean
• Saudi Arabia

67. Sickle Cell Anemia

68. Sickle Cell Anemia

69. Sickle Cell Anemia

70. Sickle Cell Anemia

71. Sickle Cell Anemia
• Pathophysiology:
• Mutation in the hemoglobin gene. It begins
with the substitution of valine for glutamic
acid at the sixth position of the beta-globin
in chromosome 11.
• Formation of HbS - under decreased O2
tension this form crescent shaped crystals
(rigid, inflexible)

72. Sickle Cell Anemia
• Pathophysiology:
• This S-shaped erythrocytes will get stuck in
capillaries
• Resulting in hemolysis & less O2 to the body
tissues
• Infraction in organs like spleen, GI tract, urinary,
heart, lungs, brain, bones, tendons & muscles
• Organ failure & multisystem diseases
Death

73. Sickle Cell Anemia
• Clinical Features:

74. Sickle Cell Anemia
• Clinical Features:

75. Sickle Cell Anemia
• Clinical Features:
Dactylitis

76. Sickle Cell Anemia
• Other Clinical Features:
• Mild jaundice
• Fever
• Headache
• Growth retardation
• Superadded bacterial infections
• Enlarged heart
• Non-healing ulcer
• Organ failure
• Vision problem

77. Sickle Cell Anemia
• Types of Crisis:
• Vaso-occlusive crisis – Occurs when
microcirculation is obstructed by Sickled
RBC and result in ischemic injury. The
major complaint is pain
• Acute chest syndrome – this is a type of
vaso-occlusive crisis that affects the lungs
& presents with chest pain, cough,
tachypnea, dyspnea, hypoxemia, fever
etc.

78. Sickle Cell Anemia
• Types of Crisis:
• Sequestration crisis – This is due to
sickled cells that block splenic outflow,
leading to the pooling of peripheral blood
in the engorged spleen resulting in
splenic sequestration.
• Aplastic crisis – can occur when the
bone marrow stops producing RBCs.

79. Sickle Cell Anemia
• Complications:
• Stroke
• Acute Chest syndrome
• Pulmonary hypertension
• Organ damage
• Blindness
• Leg ulcer
• Gall stones
• Priapism
• Pregnancy complications

80. Sickle Cell Anemia
• D/S:
• Blood test – Peripheral blood smear
• - HbS detection by
electrophoresis
• Sickling test
• Antenatal diagnosis by
–Chorionic villi sampling
–Aminocentesis
• Imaging studies

81. Sickle Cell Anemia
• Prevention:
• Genetic Counseling: If you carry the
sickle cell trait, seeing a genetic counselor
before trying to conceive can help you
understand your risk of having a child with
sickle cell anemia. They can also explain
possible treatments, preventive measures
and reproductive options.

82. Sickle Cell Anemia
• Preventing Pain Crisis:
• Consuming adequate amount of fluid to
prevent dehydration (especially during
febrile period & hot weather)
• Avoid exposure to extreme cold, exercise
or drugs that can cause acidosis
• Avoid visit to high altitude (>10,000feet)
• Avoid hypoxemia in the perioperative
period when GA is used

83. Sickle Cell Anemia
• Management:
• Management of sickle cell anemia is
usually aimed at avoiding pain episodes,
relieving symptoms and preventing
complications.
–Medications
–Pain management
–Blood transfusion
–Stem cell transplantation
–Prevention of infection

84. Sickle Cell Anemia
• Management:
• Hydration – can be corrected by oral
intake if can tolerate it.
• In severe, dehydration – IV fluids
• Accurate I/O chart maintenance is
required to prevent overload
• Analgesia – mainstay of treatment in a
pain crisis. Narcotic analgesia is most
frequently used.

85. Sickle Cell Anemia
• Management:
• Blood transfusion – exchange blood
transfusion in cases of CVA & Acute chest
syndromes.
• Oxygen supplementation – intubation &
mechanical ventilation may be required.
• Medications – Hydroxyurea. Daily
hydroxyurea reduces the frequency of
painful crises and might reduce the need
for blood transfusions and hospitalizations.

86. Sickle Cell Anemia
• Management:
• Medications – L-glutamine oral powder
(Endari). For the prevention of Acute Vaso-
occlusive Events in Children and
Adults.(FDA,2017)
• Crizanlizumab (Adakveo). Given through a vein, it
helps reduce the frequency of pain crises. (FDA,
2019)
• Voxelotor (Oxbryta). Oral drug to improve anemia
in people with sickle cell disease. works by
increasing the affinity of hemoglobin for
oxygen. (FDA, 2019)

87. Sickle Cell Anemia
• Management:
• Stem cell transplantation – Allogenic BMT
is preferred (sibling).
• Because of the risks associated with a bone
marrow transplant, the procedure is
recommended only for people, usually
children, who have significant symptoms and
complications of sickle cell anemia.

88. Sickle Cell Anemia
• Preventive care:
• Penicillin prophylaxis or amoxicillin, at
least until 5 years of age
• Immunization – pneumococcal,
meningococcal & H. influenza B vaccines.
• Life long folate supplementation
• Coping and support

89. HEMOPHILIA
• Hemophilia is a rare
disorder in which your
blood doesn't clot normally
because it lacks sufficient
blood-clotting proteins
(clotting factors).
• If you have hemophilia,
you may bleed for a longer
time after an injury than
you would if your blood
clotted normally.

90. HEMOPHILIA
• But it is the most common of the hereditary
bleeding disorders(clotting defects),
constituting nearly 90 – 95% of such cases.
• Blood contains many proteins called clotting
factors that can help to stop bleeding.
People with hemophilia have low levels of
either factor VIII (8) or factor IX (9). The
severity of hemophilia that a person has is
determined by the amount of factor in the
blood.

91. HEMOPHILIA
• Definition:
• Hemophilia is an X-linked congenital
bleeding disorder caused by a deficiency of
coagulation factor VIII (FVIII) (in hemophilia
A) or factor IX (FIX) (in hemophilia B). The
deficiency is the result of mutations of the
respective clotting factor genes.
• Incidence:
• Found in males but transmitted by female
carriers. Estimated that one in 10,000 births

92. HEMOPHILIA
• Incidence:
• Hemophilia A occurs in 1 in 5,000 live male
births. Hemophilia A is about four times as
common as hemophilia B.
• The worldwide incidence of hemophilia is not well
known, but estimated at more than 400,000
people.(A new study says more than 1125000 men
globally have hemophilia)
• Hereditary hemophilia account 80% cases where
as acquired accounts 20% due to spontaneous
mutation where family history is absent.

93. HEMOPHILIA
• Causes
• Hemophilia is caused by a mutation or change, in
one of the genes, that provides instructions for
making the clotting factor proteins needed to form
a blood clot. This change or mutation can prevent
the clotting protein from working properly or to be
missing altogether.
• These genes are located on the X chromosome.
• Males can have a disease like hemophilia if they
inherit an affected X chromosome that has a
mutation in either the factor VIII or factor IX gene.

94. HEMOPHILIA

95. HEMOPHILIA
• Types:
There are several different types of haemophilia. The
following two are the most common:
• Haemophilia A (Classic Haemophilia)
This type is caused by a lack or decrease of
clotting factor VIII (Anti Haemophilic Factor).
• Haemophilia B (Christmas Disease)
This type is caused by a lack or decrease of
clotting factor IX (Plasma Thromboplastin
Component).
• Haemophilia C: Deficiency of factor XI, the plasma
thromboplastin antecedent (PTA)

96. HEMOPHILIA
• Types:
Severity: (percentage breakdown of overall
hemophilia population by severity)
• Severe (factor levels less than 1%)
represent approximately 60% of cases
• Moderate (factor levels of 1-5%) represent
approximately 15% of cases
• Mild (factor levels of 6%-30%) represent
approximately 25% of cases

97. HEMOPHILIA
• Pathophysiology:
Due to the decreased or absence
or deficient function of blood
coagulation factors (VIII or IX)
Excessive, prolonged bleeding
or delayed clotting
Clinical features

98. HEMOPHILIA
• Signs and Symptoms:
• Common signs of haemophilia include:
• Bleeding into the joints. This can cause
swelling and pain or tightness in the joints; it
often affects the knees, elbows, and ankles.
• Bleeding into the skin (which is bruising) or
muscle and soft tissue causing a build-up of
blood in the area (called a hematoma).
• Bleeding of the mouth and gums, and bleeding
that is hard to stop after losing a tooth.

99. HEMOPHILIA
• Signs and Symptoms:
• Bleeding after circumcision (surgery performed
on male babies to remove the hood of skin,
called the foreskin, covering the head of the
penis).
• Bleeding after having shots, such as
vaccinations.
• Bleeding in the head of an infant after a difficult
delivery.
• Blood in the urine or stool.
• Frequent and hard-to-stop nose bleeds.

100. HEMOPHILIA
• D/S:
• History & clinical features
• Genetic history
• CBC, Platelet function test, clotting studies
• Imaging studies
• Antenatal diagnosis possible at 18-20 weeks of
gestation
– Foetal blood sampling
– PCR technique
– DNA/gene analysis

101. HEMOPHILIA
• Management:
• There is no cure for haemophilia. Treatment
is done to stop bleeding.
• Principle of care:
–The primary aim of care is to prevent and
treat bleeding with the deficient clotting
factor.
–Acute bleeds should be treated as quickly
as possible, preferably within two hours. If
in doubt, treat.

102. HEMOPHILIA
• Management:
• Principle of care:
–To facilitate appropriate management in
emergency situations, all patients should
carry easily accessible identification
indicating the diagnosis, severity of the
bleeding disorder, inhibitor status, type of
treatment product used, initial dosage for
treatment of severe, moderate, and mild
bleeding, and contact information of the
treating physician/clinic.

103. HEMOPHILIA
• Management:
• Comprehensive care :
–Comprehensive care promotes physical
and psychosocial health and quality of life
while decreasing morbidity and mortality.
–Comprehensive care team-
multidisciplinary in nature
–Patients should be seen by all core team
members at least yearly

104. HEMOPHILIA
• Management:
• Fitness and physical activity :
–Physical activity should be encouraged to
promote physical fitness and normal
neuromuscular development, with
attention paid to muscle strengthening,
coordination, general fitness, physical
functioning, healthy body weight, and self-
esteem.

105. HEMOPHILIA
• Management:
• Adjunctive management :
– First aid measures: Protection (splint), Rest,
Ice, Compression, and Elevation (PRICE)
may be used as adjunctive management for
bleeding in muscles and joints.
– Physiotherapy/rehabilitation
– Anti-fibrinolytic drugs (e.g. tranexamic acid,
epsilon aminocaproic acid) are effective as
adjunctive treatment for mucosal bleeds and
dental extractions

106. HEMOPHILIA
• Management:
• Adjunctive management :
– Desmopressin may increase levels
of factor VIII.
–Where clotting factor concentrates are not
available, access to safe blood
components such as fresh frozen plasma
(FFP) and cryoprecipitate.

107. HEMOPHILIA
• Management:
• Prophylactic factor replacement therapy :
–Prophylaxis is the treatment by
intravenous injection of factor concentrate
in order to prevent anticipated bleeding.
–Administration and dosing schedules.
There are two prophylaxis protocols
currently in use for which there is long-
term data:
■ The Malmö protocol: 25-40 IU/kg per

108. HEMOPHILIA
• Management:
• Prophylactic factor replacement therapy :
–dose administered three times a week for
those with hemophilia A, and twice a
week for those with hemophilia B.
■ The Utrecht protocol: 15-30 IU/kg per
dose administered three times a week for
those with hemophilia A, and twice a
week for those with hemophilia B.

109. HEMOPHILIA
• Management:
• Home therapy :
–Health education
–Home therapy allows immediate access
to clotting factor
–Home treatment must be supervised
closely by the comprehensive care team
• Pain management : Acute and chronic pain
are common in patients with hemophilia.

110. HEMOPHILIA
• Management:
• Pain management :
o application of a local anesthetic spray or cream
at the site of venous access
o Pain caused by joint or muscle bleeding care
measures include cold packs, immobilization,
splints, and crutches
o Intramuscular injection of analgesia should be
avoided.
o In Post-operative pain, IV morphine or other
narcotic analgesics can be given, followed by

111. HEMOPHILIA
• Management:
• Pain management :
o an oral opioid such as tramadol, codeine,
hydrocodone, and others. And when pain is
decreasing, paracetamol/acetaminophen may
be used.
o Avoid other NSAIDs like aspirin

112. Immune Thrombocytopenia
• Idiopathic thrombocytopenic
purpura (ITP), renamed as
immune thrombocytopenia
based on the evidence of auto-
antibody mediated consumption
of platelets, is the commonest
bleeding disorder presenting in
children.
• People with the disease have
too few platelets in the blood.

113. Immune Thrombocytopenia
• Definition:
• Is a type of thrombocytopenic
purpura defined as an isolated low platelet
count with a normal bone marrow in the
absence of other causes of low platelets. It
causes a characteristic red or purple bruise-
like rash and an increased tendency to
bleed.
• Incidence:
• Illness usually presents between 1 & 7 yr age.

114. Immune Thrombocytopenia
• Incidence:
• The annual prevalence is estimated at 5.3
per 100,000 among children.
• Worldwide, it is estimated that there are
well over 200,000 people affected by ITP.
• In India 6.4 per 100000 children
• Types:
• Acute Thrombocytopenic Purpura
• Chronic Thrombocytopenic Purpura

115. Immune Thrombocytopenia
• Types:
• Acute Thrombocytopenic Purpura:
Thrombocytopenia is termed acute if lasting
<6months; and often follows an infection and
spontaneously resolves. The majority of the
children (60-75%) are likely to have acute
ITP
• Chronic Thrombocytopenic Purpura: If the
thrombocytopenia last >6months without any
specific cause being unknown

116. Immune Thrombocytopenia
• Causes:
• Autoimmunity – development of IgG auto
antibodies
• Antecedent viral infection is common
• Drugs like penicillin, sulfonamides, heparin
• Post transfusion purpura

117. Immune Thrombocytopenia
• Pathogenesis:
• In approximately 60 percent of
cases, antibodies against platelets can be
detected. Most often these antibodies are
against platelet membrane glycoproteins IIb-
IIIa or Ib-IX, and are of the immunoglobulin
G (IgG) type.
• Platelets with antibodies on their surface are
trapped in the spleen, where they are
removed by splenic macrophages.

118. Immune Thrombocytopenia
• Pathogenesis:
• The coating of platelets with IgG renders them
susceptible to opsonization & phagocytosis
by splenicmacrophages, as well by Kupffer cells in
the liver.
• The IgG autoantibodies are also thought to
damage megakaryocytes. Recent research now
indicates that impaired production of
the glycoprotein hormone thrombopoietin, which is
the stimulant for platelet production, may be a
contributing factor to the reduction in circulating
platelets.

119. Immune Thrombocytopenia
• Clinical Features:
• Often an antecedent history of febrile illness
• The classic presentation of ITP is a
previously healthy 1-4 yr old child who has
sudden onset of generalized petechiae and
purpura
• Often there is bleeding from the gums and
mucous membranes, particularly with
profound thrombocytopenia (platelet count
<10 × 109/L).

120. Immune Thrombocytopenia
• Clinical Features:
• Classification system Depending on the
basis of symptoms and signs, but not
platelet count; ITP is classified as:
–Class 1: No symptomes
–Class 2. Mild symptoms:
Bruising and petechiae
Occasional minor epistaxis
Very little interference with daily living

121. Immune Thrombocytopenia
• Clinical Features:
• Classification system Depending on the basis of
symptoms and signs,
– Class 3. Moderate:
More severe skin and mucosal lesions
More troublesome epistaxis and menorrhagia
– Class 4. Severe:
Bleeding episodes—menorrhagia, epistaxis,
melena—requiring transfusion or hospitalization
Symptoms interfering seriously with the quality
of life

122. Immune Thrombocytopenia
• D/S:
• CBC – low platelet count
• Peripheral smear for abnormal cells
• Liver & renal function test
• Appropriate evaluation in patient with febrile
• BM examination
• Coombs (Direct) test

123. Immune Thrombocytopenia
• Treatment :
• With rare exceptions, there is usually no
need to treat based on platelet
counts. Current guidelines recommend
treatment only in cases of significant
bleeding. Treatment recommendations
sometimes differ for adult and paediatric
ITP.

124. Immune Thrombocytopenia
• Treatment :
 Acute ITP - Management consist of minimizing
the risk of severe bleeding
An initial activity limitation
Controlling active bleeding
Drugs with anti platelet function like aspirin &
anti histamine should be avoided
Platelet transfusion for severe bleeding not
responding to drug therapy (contraindicated
unless life threatening bleeding exist)

125. Immune Thrombocytopenia
• Treatment :
Acute ITP - Pharmacotherapy
Corticosteroids like prednisolone, 2
mg/kg/day for 2 – 4 weeks;
dexamethasone, 20 mg/ m² over 4 days
every 3 weeks for 4 -6 wks;
methylprednisolone, 30mg/kg/day for
three days
IVIG (IV immunoglobulin G) 1 g/kg/day for
1 or 2 days or 400mg/kg/day for 5 days

126. Immune Thrombocytopenia
• Treatment :
Acute ITP - Pharmacotherapy
Anti Rh globulin (anti D), 50 – 75 mg/kg
but this is not appropriate method of
treatment in sever cases as platelet count
doesn't improve until 48 hrs

127. Immune Thrombocytopenia
• Treatment :
 Chronic ITP - Pharmacotherapy
Mild to moderate cases do not require much
treatment. Only cases with persistently severe
thrombocytopenia or with significant mucosal
bleed need to be treated.
Methylprednisolone, 30mg/kg/day for three
days followed by 30 mg/kg for one day every
month
Dexamethasone, 20 mg/m²/day for 4 days
every month for 6 months

128. Immune Thrombocytopenia
• Treatment :
 Chronic ITP - Pharmacotherapy
Long term low dose steroids
IVIG
Anti – DIG
Spleenectomy but risk of haemorrhage. But
before spleenectomy, wait until the child is older
than 5 years of age is recommended because of
the increased risk of bacterial infection.
Pneumococcal and meningococcal vaccines are
also recommended before.
Minimum 3 yrs penicillin prophylactic therapy

129. Immune Thrombocytopenia
• Treatment :
 New Approaches:
• Drugs that boost platelet production:
Medications such as romiplostim (Nplate) and
eltrombopag (Promacta) help your bone marrow
produce more platelets.
• Other drugs. Rituximab (Rituxan, Truxima) helps
increase your platelet count by reducing the
immune system response that's damaging your
platelets.