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Autologous cd34+ cells transduced to express human adenosine deaminase


Infusion of autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes human adenosine deaminase (ADA) cDNA sequence.

Drugs List

  • autologous CD34+ cells trans to ex human adenosine deaminase dispersion for infusion 50ml bag 1-10 million cells/ml
  • STRIMVELIS 1-10 million cells/ml dispersion for infusion 50ml bag
  • Therapeutic Indications


    Severe combined immunodeficiency due to adenosine deaminase deficiency

    Treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) with no suitable human leukocyte antigen related stem cell donor.


    Busulfan 0.5 mg/kg every 6 hours intravenously for two consecutive days starting three days before treatment.

    Measure busulfan plasma levels after the first dose of each day. Reduce dose of busulfan if AUC exceeds 4000 nanograms/ml/hour.

    2 to 20 million CD34+ cells/kg as a single dose.

    If the product contains less than 2 million CD34+ cell/kg, a decision needs to be made on whether or not to proceed with the treatment due to previous treatment failures with doses below 2 million CD34+ cell/kg.

    Additional Dosage Information

    The patient is required to donate a minimum of 4 million purified CD34+ cells/kg for the treatment procedure.

    A minimum of 1 million CD34+ cells/kg should be collected from the patient as a backup at least 3 weeks prior to treatment. This is used for the rescue treatment if the treatment cannot proceed as expected.


    Autologous CD34+ cells transduced to express human adenosine deaminase is administered by intravenous infusion.

    The treatment should be prepared according to the manufacturer's instructions.


    History of gene therapy
    Patients over 65 years
    Hepatitis B
    Hepatitis C
    Myelodysplastic syndrome
    Positive HIV status

    Precautions and Warnings

    Children aged 6 to 18 years
    Children under 6 months

    Sodium content of formulation may be significant
    Premedication with antihistamine recommended
    For autologous use only
    Treatment to be administered by or under supervision of specialist
    Autoimmune disorders can occur many months after initiation of treatment
    Monitor full blood count regularly
    Monitor patient for catheter-related adverse events
    Monitor patient for signs of serious infection
    Risk of developing opportunistic infections
    Female:Barrier contraception required during & for 6 months after treatment
    Advise patient to avoid donating blood, organs, tissues or cells

    Confirm the patient's identity match the unique patient information prior to infusion.

    Patients previously positive with hepatitis C will require three sequential negative test results over a period of 4 weeks, the final test will need to be conducted within three days prior to cell harvest. Nucleic acid test results with a limit of quantification of below 15 international units/ml are required.

    Due to a potential risk of leukaemia transformation, patients require a complete blood count with differential, biochemistry and thyroid stimulating hormone annually for the first eleven years and then at thirteen and fifteen years following treatment.

    Possibility of treatment failure with older patients should be considered due to lower availability of CD34+ cells. Treatment should be reserved only when all other alternative treatment options have been considered.

    Avoid treatment if a positive test for any infectious agents listed in the EU Cell and Tissue Directive prior to bone marrow harvest.

    Pregnancy and Lactation


    Autologous CD34+ cells transduced to express human adenosine deaminase is contraindicated during pregnancy.

    The manufacturer states that limited data on exposed pregnancies are available and reproductive or developmental toxicity studies were not performed.


    Autologous CD34+ cells transduced to express human adenosine deaminase is contraindicated during breastfeeding.

    The manufacturer states that it is unknown whether autologous CD34+ cells transduced to express human adenosine deaminase are excreted in human milk. At the time of writing there are no studies on the effect of breast-fed infants following the administration of the treatment to their mothers.

    Side Effects

    Allergic dermatitis
    Anti-neutrophil cytoplasmic antibodies (ANCA)
    Autoimmune disorders
    Autoimmune hepatitis
    Guillain-Barre syndrome
    Increase in antinuclear antibodies (ANA)
    Increases in hepatic enzymes
    Smooth muscle antibody positive

    Further Information

    Last Full Review Date: October 2021

    Reference Sources

    Summary of product characteristics: Strimvelis 1-10 million cells/ml dispersion for infusion. Orchard Therapeutics (Europe) Ltd. Revised January 2021.

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    Medscape UK | Univadis prescription drug monographs & interactions are based on FDB Multilex Content

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    FDB Disclaimer : FDB Multilex is intended for the use of healthcare professionals and is provided on the basis that the healthcare professionals will retain FULL and SOLE responsibility for deciding what treatment to prescribe or dispense for any particular patient or circumstance.