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Managing APDS

The management of APDS varies from patient to patient, even within affected families, and depends on the symptoms present and disease severity.1

Current treatment is symptom-based and no guidelines exist. Polypharmacy and surgical interventions are frequently necessary to treat both the immune deficiency and immune dysregulation arms of APDS.1-4

Summary: Management Landscape For APDS*

Treatment
Effect in APDS
Proposed mechanism of
action in APDS
Antimicrobial prophylaxis
  • Reduction in respiratory tract infections 1
  • Does not address lymphoproliferation 2
Prevention of respiratory infections1
Immunoglobulin replacement therapy (IRT)
  • Reduction in respiratory tract infections 1
  • Does not appear to prevent herpesvirus infections or some sinopulmonary infections 2-4
  • Bronchiectasis can still progress 2-4
  • Does not address autoimmunity, lymphoproliferation, or lymphoma 2,3,5
Correction of antibody deficiency 1
Corticosteroids
  • Reduction in lymphoproliferation & of autoimmune cytopenias 6-8
  • Prolonged use associated with long-term toxicity 9
  • Increases susceptibility to infections 9
Inhibition of leukocyte activity and proliferation, particularly T cells9
Rituximab
  • Some benefit in the management of autoimmune cytopenias and lymphoproliferation 6,7
  • May be complicated by sustained B cell lymphopenia 6
  • Increases susceptibility to severe infection 10
Depletion of autoreactive B cells11
mTOR inhibitors
  • Reduction in lymphoproliferation 1 & infections, 12 increase in IgG, 13 reduction in IRT or steroid use, 13,14 and prevention of end organ damage12
  • Effects on bowel inflammation & cytopenia are mixed14
  • Difficulty dosing, adverse events, and compliance are challenges to treatment 6,13-16
Reduction in mTOR hyperactivation1
Hematopoietic stem cell transplantation (HSCT)
  • Reduction in respiratory & herpes infections, lymphoproliferation, & autoimmunity 1
  • Serious complications and complex conditioning regimens 17
  • May not correct non-immunological manifestations such as kidney disease 17
  • Paucity of long term data 17
Replacement of leukocytes affected by PI3Kδ

Summary: Management Landscape For APDS*

Treatment

Antimicrobial prophylaxis

Effect in APDS

  • Reduction in respiratory tract infections 1
  • Does not address lymphoproliferation 2

Proposed mechanism of action in APDS

Prevention of respiratory infections 1

Treatment

Immunoglobulin replacement therapy (IRT)

Effect in APDS

  • Reduction in respiratory tract infections 1
  • Does not appear to prevent herpesvirus infections or some sinopulmonary infections 2-4
  • Bronchiectasis can still progress 2-4
  • Does not address autoimmunity, lymphoproliferation, or lymphoma 2,3,5

Proposed mechanism of action in APDS

Correction of antibody deficiency1

Treatment

Corticosteroids

Effect in APDS

  • Reduction in lymphoproliferation & of autoimmune cytopenias 6-8
  • Prolonged use associated with long-term toxicity 9
  • Increases susceptibility to infections 9

Proposed mechanism of action in APDS

Inhibition of leukocyte activity and proliferation, particularly T cells 9

Treatment

Rituximab

Effect in APDS

  • Some benefit in the management of autoimmune cytopenias and lymphoproliferation 6,7
  • May be complicated by sustained B cell lymphopenia 6
  • Increases susceptibility to severe infection 10

Proposed mechanism of action in APDS

Depletion of autoreactive B cells11

Treatment

mTOR inhibitors

Effect in APDS

  • Reduction in lymphoproliferation 1 & infections, 12 increase in IgG, 13 reduction in IRT or steroid use, 13,14 and prevention of end organ damage 12
  • Effects on bowel inflammation & cytopenia are mixed 14
  • Difficulty dosing, adverse events, and compliance are challenges to treatment6,13-16

Proposed mechanism of action in APDS

Reduction in mTOR hyperactivation 1

Treatment

Hematopoietic stem cell transplantation (HSCT)

Effect in APDS

  • Reduction in respiratory & herpes infections, lymphoproliferation, & autoimmunity 1
  • Serious complications and complex conditioning regimens 17
  • May not correct non-immunological manifestations such as kidney disease 17
  • Paucity of long term data 17

Proposed mechanism of action in APDS

Replacement of leukocytes affected by PI3Kδ
Showing 1 to 6 of 6 entries
Table modified from Coulter TI, Cant AJ. Front Immunol. 2018;9:2043

* The treatments listed represent current practice as denoted in the literature. The information provided does not replace medical advice. None of the treatments listed above have been FDA-approved specifically for APDS.

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PI is also referred to as an Inborn Error of Immunity (IEI).

References:

1. Coulter TI, Cant AJ. Front Immunol. 2018;9:2043. 2. Elgizouli M, et al. Clin Exp Immunol. 2016;183(2):221-229. 3. Crank MC, et al. J Clin Immunol. 2014;34(3):272-276. 4. Kannan JA, et al. Ann Allergy Asthma Immunol. 2015;115(5):452-454.

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