Indications for cord blood

collection in
haemoglobinopathies

Dr Jo Howard
Guy’s and St Thomas’ NHS
Trust
• Are cord blood transplants in the
haemoglobinopathies effective?
• Is cord blood banking in the
haemoglobinopathies feasible?
• Who should have cord blood banked?
Are cord blood transplants in
the haemoglobinopathies
effective?
• YES
• Josu de la Fuente
• Cord blood stem cell transplantation for
haemoglobinopathies
- Fernando O Pinto and Irene Roberts
- Matched sibling CBT as effective as BMT
- Dose must be adequate
• Unrelated cord transplantation is
experimental procedure
Is cord blood banking in the
haemoglobinopathies feasible?
• US experience
– 1600 samples over 6 years, 96 (6%) for
thalassaemia, 450 (28%) for sickle cell
– In thalassaemia, 32 (33%) were HLA
compatible and 14 (44%) received CBT
– In sickle, by 2003, 163 CB units, 4 (2%)
used for CBT. All engrafted, ¾ have long
term survival.
– By 2005, 8 CBT (2% of units)
Is cord blood banking in the
haemoglobinopathies feasible?

• UK experience
– 44 units collected from families with
haemoglobinopathies (36 thalassaemia)
– 7 of units for thalassaemia had been
used (20%)
– All patients alive and well (1 had
secondary graft rejection)
– No units for sickle had been used

Smythe et al. Stem Cells 2007;25:2087-2093

Reasons for low use of CBT
• Low uptake of cord blood banking
– Cost
– Lack of awareness
– Social/family reasons
– Negative views of CBT by patients or
physicians
• Low use of banked cords
– HLA incompatibility
Dilemma 1
• We need to ‘investigate the reasons both
for low banking rate and low usage rate
before formulating policies… about
systematic collection of CB’
• We are being asked by patients to provide
this service, and if we do not provide on
NHS they not get service, or have to use
private providers
• PCTs will not fund individual patients
without hospital protocols
Draft Protocol
Eligibility Criteria
• Only applies to directed sibling
donations of cord blood for
banking for potential CBT in future
A) An existing sibling could be
treated by allogeneic transplant
B) An earlier birth from the same
parents indicated an inherited
condition that could result in a
future infant needing an allogeneic
transplant
Dilemma 2

• Should we include
C) prenatal screening has revealed the
presence of a genetic condition that
may be treated by allogeneic
transplantation in a future infant
Eligible conditions

• Inherited anaemias
– B thalassaemia, Sickle Cell Disease
• Haematological malignancies
• Immunodeficiency
• Enzyme deficiency
• Others eg aplastic anaemia
Referral process

• Patient may present to

– Obstetricians
– Midwives
– Paediatricians
– Haematologists
– Community haemoglobinopathy nurses
Referral process

• Initial referral goes to ‘Home from

Home Birth Centre Consultant
Midwife’
• They will co-ordinate process and
referral to SCI-NBS
• <30/40 weeks gestation
• If concerns about eligibility refer to
‘Trust Working Party on Cord Blood
Donation’
Referral process
• All referrals agreed by

A) Consultant Obstetrician –
responsible for medical review of the
mother
B) Consultant Paediatrician or
Haematologist or Clinical Geneticist –
has responsibility for affected sibling
or can advise about liklihood of
transplantation in a future sib
Trust Working Party on Cord
Blood Donation
• Home to Home Birth Centre
Consultant Midwife
• Consultant Obstetrician
• Consultant Paediatrician
• Consultant Haematologist
• Manager
Trust Working Party on Cord
Blood Donation
• Will decide on eligibility for directed
cord blood donation
• Will review initial results from NBS on
cord blood collected and deciding on
storage
• Yearly review of stored cords
• Will keep directed cord blood
protocol up to date
Other issues
• Numbers
– 2-3 women who already have affected
children
– 6 -10 presenting via prenatal screening
• Funding
– PCT
• How can couples be informed
– Community team as part of prenatal
screening
• New UK guidelines