Name: Aqneursa (levacetylleucine) in Ataxia-telangiectasia: Immunoglobulin replacement and infection prophylaxis
Published: 2026-10-02T18:22:10-04:00

Product information and disease description Place of product in therapy Approaches to treatment Current treatment options and standard of care

Immunoglobulin replacement and infection prophylaxis

4 evidence topics · 4 sources

Guideline indications for immunoglobulin replacement

“Immunoglobulin replacement therapy, aiming at a serum immunoglobulin-G >6g/L, is indicated in all patients with a severe immunoglobulin-G deficiency (serum immunoglobulin-G <4g/L)” (opens the source at this quote in a new tab)

“Immunoglobulin replacement therapy is indicated in individuals with severe IgG deficiency and in those with the hyper IgM phenotype.” (opens the source at this quote in a new tab)

“Immune replacement therapy is considered when hypogammaglobulinemia and/or specific antibody deficiency is present together with a history of recurrent respiratory infections or established bronchiectasis, or when other preventive measures such as vaccinations and prophylactic antibiotics do not result in clinical improvement of the RTI symptoms irrespective of the severity of the immunological abnormalities.” (opens the source at this quote in a new tab)

Proportion of patients who receive immunoglobulin replacement

“About 15% of individuals with A-T will require Immunoglobulin replacement.” (opens the source at this quote in a new tab)
“Subcutaneous administration has shown to be both efficacious and acceptable to patients on replacement therapy.” (opens the source at this quote in a new tab)

“Hence there is wide variation in the proportion of patients who are on replacement treatment (table 2).” (opens the source at this quote in a new tab)
“If Ig therapy is considered, most centres administer subcutaneous Ig, as is currently recommended for severe immundeficiencies [29–31].” (opens the source at this quote in a new tab)

Retrospective series	Receiving immunoglobulin therapy (%)
80 participants	“12.5”
100 participants	“13”
15 participants	“60”
240 participants	“34.8”
44 participants	“15.9”
100 participants	“18.8”

Evidence base for prophylactic antibiotics and immunoglobulin replacement

“No placebo-controlled trials have been performed with regards to prophylactic antibiotic therapy or immune replacement therapy.” (opens the source at this quote in a new tab)
“A high proportion (74%) of children with A-T are prescribed prophylactic antibiotics [24] on inconsistent and non-evidence-based criteria.” (opens the source at this quote in a new tab)

Guideline position on prophylactic antibiotics

“Antibiotic prophylaxis may be indicated in patients with frequent or severe infections, and should be advised in immunoglobulin-G deficiency or specific polysaccharide antibody deficiency.” (opens the source at this quote in a new tab)
“Antibiotic prophylaxis has not been studied in ataxia-telangiectasia” (opens the source at this quote in a new tab)

“Prophylactic antibiotic treatment is considered when the burden of respiratory infections is high, if there is associated specific polysaccharide antibody deficiency and when there is established bronchiectasis.” (opens the source at this quote in a new tab)

“There is no data to recommend, or otherwise, prophylactic antibiotics, but most children are recommended prophylactic azithromycin by the immunologists.” (opens the source at this quote in a new tab)