2015 WASN Spring Conference
5C: Von Willebrand Disease and Other Bleeding Disorders in School Aged Children
Objectives
Von Willebrand
Disease and Other
Bleeding Disorders in
School Aged Children
Jessie Pindilli,BSN, RN
Program Coordinator
UW Hospital & Clinics
Comprehensive Program for Bleeding
Disorders
 Describe normal hemostasis
 Describe Von Willebrand Disease and
Hemophilia
 Identify signs and symptoms of bleeding
disorders, particularly in female students
 Describe what to do in an emergency
 Recognize potential barriers that children with
a bleeding disorder may face at school
April 24, 2015
Comprehensive Care at Hemophilia
Treatment Centers (HTC)
 Team members all have expertise and experience in treating
bleeding disorders
– Core members include hematologist, nursing, social work,
and physical therapy
– Comprehensive, interdisciplinary care model
 Access to clotting factor concentrates
 Over 130 HTCs throughout the U.S. & territories
 Rate of hospital stays for bleeding-related events among
patients seen in HTC’s was 40% lower than the rates of
patients seen in other care facilities (Soucie et al, 2000)
 Patients seen in HTCs are 40% less likely than those seen
elsewhere to die of a hemophilia related complication. (Soucie
et al, 200)
 Wisconsin: Milwaukee (Children’s Hospital of Wisconsin),
Madison (University of WI Hospital and Clinics), Green Bay
(Hemophilia Outreach Center), and La Crosse (Gundersen
Clinic)
Normal
Hemostasis
Stages of Hemostasis
Three Distinct Phases:
1. Primary Hemostasis
• Occurs after damage to the vessel wall
• Involves vasoconstriction and adhesion of platelets
on subendothelial vessel wall.
• Further platelets aggregate to form a platelet plug
2. Secondary Hemostasis
• Activation of the coagulation system, leading to the
generation of fibrin strands, which are laid down
between platelets and reinforce the platelet plug.
• Clotting factor activation leading to fibrin formation
3. Fibrinolysis
• Breakdown of clot
Abnormal
Hemostasis due to
incomplete fibrin
clot formation
Measure of Hemostasis
Clot Dissolution
(Fibrinolysis)
Clot Formation
(Coagulation)





PT
aPTT
Thrombin Time
Fibrinogen
Individual Clotting
Factors




FDP
D-Dimer
HMWK
Prekallikrein
1
2015 WASN Spring Conference
5C: Von Willebrand Disease and Other Bleeding Disorders in School Aged Children
“Simplified”
Effects of Factor Deficiencies on
aPTT and PT
 aPTT Prolonged, PT normal
– Deficiency of intrinsic pathway – VIII, IX, XI, XII
 PT Prolonged, aPTT normal
– Deficiency of extrinsic pathway factor - VII
 Both PT and aPTT prolonged
– Deficiency of common pathway factors –
Fibrinogen, II, V, X
– Multiple factor deficiencies
Inheritance of Hemophilia
Hemophilia
 Hemophilia A
– Factor VIII Deficiency
– 1 in 5,000 live male births in US
 Hemophilia B
– Factor IX Deficiency
– 1 in 30,000 live male births in US
 Approximately 20,000 affected in the U.S.
 X-linked, recessive inheritance
– Primarily affects males (very rarely females)
 30% are spontaneous mutations
If mother is a carrier of hemophilia…
 Daughter has 50% chance of
becoming a carrier
 Son has 50% chance of having
hemophilia
If father has hemophilia …
 All daughters will be obligate
carriers
 Sons will be unaffected
National Hemophilia Foundation Steps for Living
www.stepsforliving.hemophilia.org
Internet encyclopedia of
health and science
Severity
 Severity of disease
depends upon amount of
circulating clotting factor
(Normal = 50-150%)
– Mild (5-50%)
– Moderate (1-5%)
– Severe (<1%)
 Amount of baseline
circulating factor remains
constant throughout life
and is constant within
families
Treatment advances have
dramatically improved life expectancy
 Life expectancy
– 7.8 years in the 1930s
– 74 years (if HIV-negative) from 1990 to 2001
 15% of the total population of patients with
hemophilia are 45+ years old
Centers for Disease Control and Prevention. Summary Report of UDC Activity: National.
2
2015 WASN Spring Conference
5C: Von Willebrand Disease and Other Bleeding Disorders in School Aged Children
Clotting Factor Infusion
 Almost all severe hemophiliacs, and many mild and
moderate hemophiliacs, learn to infuse at home for
convenience
 Parents of toddlers and boys as young as age 7 begin
to learn home infusion
 Prefer morning infusions so factor is at “highest
strength” during the day when most active
 Peripheral Vein vs. Infusaport
– Infusaport often used to facilitate prophylaxis
beginning in toddlers
– Prefer not to leave accessed, especially while at
school due to increased risk of serious infection
– With any fever over 100.5 must rule out port
infection with blood cultures
Most Common Complication: Joint
bleeding
 Pediatric patient with untreated bleed to the
knee
Special Considerations in Mild-Moderate
Hemophilia
 Bleeding generally related to a procedure or
significant injury
 Sometimes less familiar with signs of a bleed or
how to treat
– Reporting of injuries or procedures is often
delayed
 Less likely to perform self infusion
 Frequently diagnosed later in life when they
face bleeding challenges
von Willebrand Disease (VWD)
 Deficiency of Von Willebrand Factor
– Clotting protein that binds with Factor VIII
– Prevents degradation of Factor VIII until it is
needed during coagulation- known as “carrier
protein” in plasma for Factor VIII.
 Most Common Bleeding Disorder
– affects 1-2% of general population
 Affects Males and Females Equally
– Most commonly autosomal dominant
inheritance (only need to inherit one of the
affected chromosomes to get the disease)
– Rarely autosomal recessive inheritance
People with hemophilia
bleed LONGER NOT
FASTER
Types of von Willebrand Disease
 Type 1 = mild
• Decreased amount of Von Willebrand Factor
• VWF Activity 30-50% = very mild bleeding,
<20%=moderately severe bleeding
• Most common
 Type 2 = qualitative
• VWF doesn’t work correctly
• Subtypes 2A, 2B, 2M, 2N
 Type 3 = severe
• Very low or No VWF
• <5% of cases
• Bleeding pattern very similar to hemophilia
 Acquired
• Rare
• Occurs late in life, r/t autoimmune complications
3
2015 WASN Spring Conference
5C: Von Willebrand Disease and Other Bleeding Disorders in School Aged Children
Platelet Function Defects
Desmopressin (DDAVP)
 Generally mucosal bleeding
 Treatments
– Platelet transfusions
– DDAVP, Antifibrinolytics
– Estrogen for menorrhagia
 Platelet Storage Pool Disorders
– Bleeding is typically mild, related to trauma and
invasive procedure
 Bernard Soulier
(very rare)
– Large, abnormal platelets & low platelet count
– Bleeding can be very severe
 Glanzmann’s Thrombasthenia
– Decreased Platelet aggregation
– Bleeding can be very severe
(very rare)
 Synthetic Anti-diuretic Hormone
– Common side effects include flushing & headache
– Risk for water retention, hyponatremia
– Limit fluid intake x24 hours after taking
 STIMATE
– Intranasal Desmopressin
– >50 kg =1 spray to each nostril
<50 kg = 1 spray to 1 nostril
– once q24 hrs for a max of 3 consecutive days
– Not readily available through local pharmacies, usually need
min 24 hrs notice
 IV DDAVP
– 3 mcg/kg IV prior to a procedure or in response to a bleed
 Von Willebrand factor concentrate
– Given intravenously. Contains factor VIII and von Willebrand
factor.
– If patient does not respond to Stimate or for certain type 2
and all of type 3 vWD.
Bleeding with VWD
Antifibrinolytics
 Prevent lysis of clots
 Aminocaproic Acid (Amicar) Tranexamic
Acid (Lysteda)
 Prescription
– Tablet, solution or IV
 Every 6-8 hours for minimum of 5 days
after bleed
Why does my nose bleed? The
answer it not always simple.
Reference: Berkowitz, C. (2008). Pediatrics: A
Primary Care Approach. Los Angeles: Saunders
Company. Images via google.
 Mucous membrane bleeding is most common, as vWF
is essential for primary hemostasis
– Nosebleeds
– Mouth bleeds
– Bruising
– Prolonged oozing from superficial cuts
– Bleeding after trauma or surgery
– Prolonged/ heavy menstrual bleeding
 Joint and muscle bleeds are less common than in
hemophilia except severe Type 3 and some Type 2
– Treat with RICE and von willebrand factor clotting
factor concentrate as indicated
How to treat mucosal bleeding
 Nosebleeds
– Same as any other child with a nosebleed
– Tilt head forward, pressure (full 10 minutes), ice
– If bleeding has not stopped after 10 minutes, repeat the
pressure. If persists, call your doctor or go to emergency
room. Stimate may be used if school has permission.
– Prevention: Cool mist humidifier in bedroom, Saline gel
(AYR)
– Urgent QR (OTC) powder
– Afrin OTC (Oxymetazoline), soak cotton balls
– Do not stuff tissues or things in the nose to stop the
bleeding
– May need evaluation for cauterization if frequent
 Mouth Bleeding
– Soft, cool or lukewarm diet until area is fully healed
– No straws, gum or smoking
– Topical thrombin for frenulum or tongue laceration
4
2015 WASN Spring Conference
5C: Von Willebrand Disease and Other Bleeding Disorders in School Aged Children
Soft Tissue/Superficial Bleed
 Soft Tissue
– Easy bruising with or without hematomas
– Generally do not require aggressive
treatment other than rest and ice if they do
not threaten function and mobility
 Superficial Cuts & Scrapes
– Treatment is typical first aid
Life & Limb Threatening Bleeds






Intracranial
Spinal Cord
Oropharynx
Gastrointestinal/Intra-abdominal
Ocular
Limb compartments/Iliopsoas
Call 911, parent, HTC (follow school plan for
emergency notification)
Require IMMEDIATE treatment with factor
concentrate
Female Students
Normal Periods
 Issues with menorrhagia
 Average age in US: 12 years
– Too early: before 8 years
– Too late: after 15 years
 Average cycle length: 21-35 days
– Too often: under 21 days
– Not often enough: greater than 35 days
 Average cycle duration: 7 days or less
Hormones
When is it too much?
 Menorrhagia
– Greater than 80 ml/cycle (average 25-80 ml)
– Greater than 7 days of menstruation
5
2015 WASN Spring Conference
5C: Von Willebrand Disease and Other Bleeding Disorders in School Aged Children
How much is too much?
Signs of menorrhagia
 Have a menstrual flow that soaks through one or more
pads or tampons every hour for several hours in a
row. (Hall passes!)
 Need to double up on pads to control your menstrual
flow.
 Need to change pads or tampons during the night.
 Have menstrual periods lasting more than 7 days.
 Have a menstrual flow with blood clots the size of a
quarter or larger.
 Have a heavy menstrual flow that keeps you from
doing the things you would do normally.
 Have constant pain in the lower part of the stomach
during your periods.
 Are tired, lack energy, or are short of breath
Prevalence of Bleeding Disorders in
Adolescents with Menorrhagia





von Willebrand Disease
Platelet Dysfunction
Factor XI Deficiency
Low Factor VIII
Thrombocytopenia
5-36%
2-44%
< 1-4%
~ 8%
13-20%
Resources for Bleeding Disorders in
Women
 http://www.fwgbd.org
 CDC – Screening Tool for Identifying Women with
Menorrhagia for Testing for Underlying Bleeding
Disorder
Treatment for menorrhagia secondary
to bleeding disorder
 Combined oral contraceptives used to treat
Menorrhagia
– usually monophasic
– often require high doses or continuous cycling
 Other contraceptive methods known to reduce amount
of menstrual
– Mirena IUD, Implanon, Depo-Provera
 DDAVP (Stimate)
– max of 3 consecutive days, consider treatment
before bed to minimize side effects)
 Antifibrinolytic agents
– Lysteda 1300 mg TID x5 days during menstruation
 Iron supplementation PRN
Acute Illness, Injury and Invasive
Procedure
 Conditions that wouldn’t normally cause a
person to bleed may put a hemophiliac at risk
for a bleed and require treatment
– e.g. coughing and pharyngitis
 Invasive procedures including dental work
other than a cleaning generally require pretreatment to prevent bleeding
 Increased metabolic states will increase factor
requirements
 May have delayed wound healing with scaring
6
2015 WASN Spring Conference
5C: Von Willebrand Disease and Other Bleeding Disorders in School Aged Children
Other Treatments
 RICE
– Rest
– Ice (15-20 minute intervals)
– Compression (Pressure for 15 min)
– Elevation
 Pain Management
– Tylenol, Avoid NSAID’s & Aspirin
– Celebrex & Opioids per MD discretion
 Typical first aid for cuts and scrapes and minor bruises
 Mild Hemophilia A - Stimate (Intranasal DDAVP)
Exercise
 Students with a bleeding disorder
are encouraged to be active and
should participate in gym and
activities
 Staying active improves joint
strength and helps maintain
normal weight
 Avoid high contact sports such
as football, hockey, wrestling,
boxing and power lifting
 Highly recommended activities are
strength training, swimming,
bicycling, jogging, golf, dancing,
hiking, bowling and aerobic or
cardiovascular training
Sports & Activities
 A child with a bleeding disorder should :
– Do warm up exercises before playing and cool
down and stretching exercises after playing
 A child with a bleeding disorder should not:
– Follow the “no pain, no gain” philosophy
– Hide a bleed or injury
– Exercise when in pain
• The child should stop an exercise if it hurts a
muscle or joint.
– Exercise when healing from an injury
• Injuries should have adequate time to heal. The
HTC can help you to know when it’s safe to
resume an activity
Preventative Health
 Dental care
– Twice yearly visits
– Brush and Floss twice daily (continue flossing
even with gum bleeding initially)
 Hepatitis A & B immunization
 Safety & injury prevention
– Helmets, seat belts, etc.
 Physical activity & nutrition to maintain healthy
weight
– Being overweight will increase burden on
joints
 Good source of iron in the diet and/or iron
supplementation
 Medic Alert Emblem
School Attendance
 Goal is for students to be at school with
minimal absences related to their bleeding
disorder
 For children aged 5-17 years, average annual
school absenteeism due to hemophilia was
4.7 days
 Parents of affected children miss 3.2 days of
work annually
 IEP
Hemophilia Utilization Group Study (HUGS)
7
2015 WASN Spring Conference
5C: Von Willebrand Disease and Other Bleeding Disorders in School Aged Children
School Awareness of
Social/Emotional Needs
 Education from HTC available to overcome
fear due to lack of knowledge
 Possible for student to have increased anxiety,
frustration due to limitations with condition
 Avoid identification of disability- focus on what
the student can do
Key Points
 People with bleeding disorders bleed longer
not faster
 Children with bleeding disorders should
participate in all school activities except
contact sports
 Female students are disproportionately
affected by von Willebrand Disease and
Platelet function defects
 Early treatment of bleeding episodes is critical
 Collaborate with Hemophilia Treatment Center
Staff
References
 Manco-Johnson, et. al. (2007). Prophylaxis vs
Episodic Treatment to Prevent Joint Disease in Boys
with Severe Hemophilia: New England Journal of
Medicine
 National Hemophilia Foundation (2000), von
Willebrand Disease Just the FAQs
 National Hemophilia Foundation. (2005) Playing It
Safe: Bleeding Disorders, Sports and Exercise
 Ramot. (2007). Toxicol Pathol Drug Induced
Thrombosis Ramot. Experimental , Clinical, and
Mechanistic Conditions.
 Soucie JM, et al. Mortality among males with
hemophilia: relations with source of medical care.
Blood 2000;96:437-42
Secondary Education & Career Planning
 Most young people with a bleeding disorder will be
able to pursue a normal education and career
– In most states having Hemophilia alone will not
qualify a person for disability
 Generally unable to pursue careers with physical
performance testing
– e.g. military, law enforcement, firefighting
 Physical, manual labor can present challenges for a
person with a bleeding disorder
 Consider the potential benefits available
– Health Insurance is a must, Good health insurance
may take priority over salary
– Best to be employed by a larger company (>100
employees)
 Many scholarships specific to students with bleeding
disorders are available
References
 Centers for Disease Control. Hemophilia.
http://www.ced.gov/ncbddd/hemophilia/HTC.html
 Centers for Disease Control and Prevention. Summary
Report of UDC Activity: National. Available at
https://www2a.cdc.gov/ncbddd/htc/web/UDC_Report/
UDC_Report/asp April 1 2012
 Dunn, A.L., Abshire, T.C. (2004) Recent advances in
the management of the child who has hemophilia.
Hematology Oncology Clinics of North America, (18)
1249-1276.
 Hemophilia in Pictures Presentation. World Federation
of Hemophilia. www.wfh.org
 MASAC Document #205 MASAC Recommendations
Concerning Products Licensed for the treatment of
hemophilia and other bleeding disorders
References
 Soucie JM, Symons J, Evatt B, Brettler D, Huszti H,
Linden J and the Hemophilia Surveillance System
Project Investigators. (2001). Home-based factor
infusion therapy and hospitalization for bleeding
complications among males with hemophila.
Haemophilia 7:198-206.
 World Federation of Hemophilia. Hemophilia of
Georgia. Protocols for the treatment of Hemophilia
and Von Willebrand Disease 3rd edition
 World Federation of Hemophilia. (2009) What are
Inhibitors?
 Wulff K, Zappa S, Womack, M and the Nursing Group
of Hemophilia Region VI. (1999) Emergency Care for
Patients with Hemophilia: An Instructional Manual for
Medical Professionals.
8
2015 WASN Spring Conference
5C: Von Willebrand Disease and Other Bleeding Disorders in School Aged Children
References
 White E, Menga Y, The Student with Hemophilia.
www.hemophilia.org Accessed March 10 2012
 Zappa S, Lacasse L, Jacobson R, Pucell S and Wulff
K. Emergency Care for Patients with von Willebrand
Disease: An Instructional Manual for Medical
Professionals
Please don’t hesitate to contact me with any
questions
jpindilli@uwhealth.org
Resources
National Hemophilia Foundation Steps for Living
www.stepsforliving.hemophilia.org
World Federation of Hemophilia
“Hemophilia in Pictures”
www.wfh.org
9