Name: Aqneursa (levacetylleucine) in Ataxia-telangiectasia: Care management intervention strategies
Published: 2026-10-02T18:22:10-04:00

United Kingdom specialist center model for children

“Once a diagnosis of A-T has been made the patient should be referred to the appropriate national specialist centre for a detailed multi-disciplinary assessment.” (opens the source at this quote in a new tab)
“Clinics are held over two days (Thursday & Friday), six times a year. At each clinic, six children are seen on an outpatient basis.” (opens the source at this quote in a new tab)
“Patients are usually recalled to the clinic every two or three years.” (opens the source at this quote in a new tab)

Local care between specialist center visits

“We also recommend that each child has a local named key worker, and a local named lead professional” (opens the source at this quote in a new tab)
“Respiratory care: A respiratory review at least every three months, and more frequently if the child is unstable.” (opens the source at this quote in a new tab)
“Neurology: A paediatric neurodisability review at least every 6 months, and more frequently if the child is unstable.” (opens the source at this quote in a new tab)

National adult service specification in England

“offer a multi-disciplinary review for patients with AT on an annual basis which is individualised to their specific requirements” (opens the source at this quote in a new tab)
“minimise hospital admissions and the requirement for unplanned medical intervention” (opens the source at this quote in a new tab)
“Patients will generally stay under the care of Papworth for their whole lifetime.” (opens the source at this quote in a new tab)

Multidisciplinary clinics in other countries

“Several multidisciplinary clinics across Europe (the UK, Netherlands, Norway, France and Germany) and elsewhere (Israel, Baltimore (MD, USA) and Brisbane (Australia)) manage patients with A-T.” (opens the source at this quote in a new tab)

Recommended surveillance

System or concern	Evaluation	Frequency
Neurologic	“Eval by neurologist for response to therapy for existing findings & development of new findings”	“Annually”
Immunodeficiency	“By immunologist (immunoglobulin levels, white blood cell count)”	“Annually”
Pulmonary disease	“Lung function testing (by spirometry)”	“Annually for persons w/evidence of disease at time of initial diagnosis”
Malignancy, laboratory	“Blood count & smear, immunoglobulin levels, M protein, LDH, IgM”	“Annually”
Malignancy, abdomen	“Ultrasound exam of abdomen in adulthood”	“Annually”
Malignancy, breast	“Breast MRI in females”	“Annually starting at age 25 yrs”
Diabetes	“Diabetes screening: urinalysis, fasting blood glucose concentration, Hgb A1c”	“Annually in adults”
Feeding and nutrition	“By SLP/nutritionist”	“Annually”

Avoidance of ionizing radiation

“Ionizing radiation (x-ray and gamma ray) is contraindicated, due to increased sensitivity” (opens the source at this quote in a new tab)
“Radiation therapy is contraindicated because increased radiosensitivity may lead to very severe complications” (opens the source at this quote in a new tab)

“Radiotherapy is strongly discouraged.” (opens the source at this quote in a new tab)
“Diagnostic radiographic examinations should be avoided” (opens the source at this quote in a new tab)

“According to this approach, a single X-ray can be performed for diagnostic purposes such as diagnosis of a chest infection or scoliosis. However, repeated X-rays should be avoided.” (opens the source at this quote in a new tab)

Vaccination guidance

“Inactivated vaccines are safe in ataxia-telangiectasia and are recommended as part of routine childhood vaccination programmes, except for patients who are on immunoglobulin replacement therapy. Annual vaccination with inactivated influenza vaccine is recommended for all patients, including those receiving immunoglobulin replacement therapy.” (opens the source at this quote in a new tab)
“Live oral polio vaccine should not be administered.” (opens the source at this quote in a new tab)

“Flu vaccines, 13-valent pneumococcal conjugate, and 23-valent pneumococcal polysaccharide vaccines should be given to all individuals with A-T.” (opens the source at this quote in a new tab)
“Rubella vaccination should be avoided in individuals with severe immunodeficiency as it can possibly increase the risk for granulomas” (opens the source at this quote in a new tab)

“Yellow fever vaccine should not be used in any patients with A-T.” (opens the source at this quote in a new tab)

Surgery and anesthesia

“Patients who have to undergo surgery should preferably be prepared, monitored, and operated in a dedicated ataxia-telangiectasia clinic.” (opens the source at this quote in a new tab)

Pregnancy testing and contraception during levacetylleucine treatment

“For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with AQNEURSA.” (opens the prescribing information at this quote)
“Advise females of reproductive potential to use effective contraception during treatment with AQNEURSA and for 7 days after the last dose if AQNEURSA is discontinued” (opens the prescribing information at this quote)
“The decision to continue or discontinue AQNEURSA treatment during pregnancy should consider the female’s need for AQNEURSA, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease.” (opens the prescribing information at this quote)

Monitoring with concomitant P-glycoprotein substrates

“Monitor more frequently for P-gp substrate related adverse reactions when used concomitantly with AQNEURSA.” (opens the prescribing information at this quote)

Missed doses

“If a dose of AQNEURSA is missed, skip the missed dose and take the next dose at the scheduled time. Do not take 2 doses at the same time to make up for a missed dose.” (opens the prescribing information at this quote)

Clinical scales available to document neurological status in practice

“We found a strong correlation between A-T NEST and the established SARA score, validating the use of A-T NEST and SARA in capturing the natural history of A-T patients.” (opens the source at this quote in a new tab)