Tolerability and Efficacy

PID refers to a large heterogeneous group of disorders that result from mostly inherited defects in the development and/or function of the immune system.1 Almost 500 different PIDs have been described so far, with more than 6 million people affected around the world.2 Around 60% of all PID cases are associated with hypogammaglobulinemia due to impaired antibody production.3 Whilst it is estimated that most of PIDs could be easily diagnosed with simple and inexpensive blood tests, many PIDs remain underdiagnosed on a global scale, leaving people affected at high risk of infections, severe autoinflammation, autoimmunity, allergy, and malignancy.4,5
IVIg replacement therapy can help address the key needs of patients with PIDs, such as:
↓ number of severe infections6,7
↓ days of antibiotic use6
↓ rate and duration of hospitalisation6,7
↑ quality of life7,8

ITP is an autoimmune disorder characterised by a low platelet count (<100 × 109/L) with an increased risk of bleeding.9 Although most patients produce antibodies to specific platelet membrane glycoproteins, the trigger for production of autoantibodies against platelets is currently unknown.9 Data on the incidence and prevalence of ITP are scarce.9 The National Organisation of Rare Diseases (NORD) estimates that affected people amount to over 200,000 people worldwide.10 Thrombocytopenia can impact many aspects of patients’ life. Symptoms are variable, and go from no to mild bleeding, post-traumatic bruising, spontaneous subcutaneous bleeding or in some rare cases life-threatening bleeding.11 Patient concerns usually include physical symptoms, social limitations due to the unpredictability of episodes and fear of bleeding in public, psychological effects and fatigue.11 Whilst treatment is typically initiated with corticosteoids, these often lead to lack of long-term responsiveness and reduced tolerability adding to the burden of the disease.9

Immunomodulation with IVIg is recommended in patients with bleeding or at high risk of bleeding, who require a surgical procedure or are unresponsive to prednisolone. IVIg is generally well tolerated9 and can induce recovery of platelet counts within a short time, providing therapeutic advantage when a rapid increase in platelets is required for patients at risk of critical bleeding.9,12

CIDP is a neurological disorder associated with an immune-mediated response directed primarily against the peripheral nervous system.13 CIDP manifests with peripheral nerve demyelination.14 Typical CIDP begins with paraesthesia and weakness in the distal limbs as well as difficulty with walking.14 The clinical examination shows progressive symmetric proximal and distal muscle weakness, sensory loss, and decreased or absent deep tendon reflexes.15 The disease course is steadily progressive for more than 8 weeks, but can be relapsing-remitting.15 However, the disease can also manifest with different phenotypes; these are usually identified as CIDP variants because they share the common features of demyelination and response to immune therapy and they include:15

• Distal CIDP: distal sensory loss and muscle weakness predominantly in lower limbs

• Multifocal CIDP: sensory loss and muscle weakness in a multifocal pattern, usually asymmetric, upper limb predominant, in more than one limb

• Focal CIDP: sensory loss and muscle weakness in only one limb

• Motor CIDP: motor symptoms and signs without sensory involvement

• Sensory CIDP: sensory symptoms and signs without motor involvement

CIDP is considered an orphan disease and prevalence rates hugely vary in different geographical regions,14 from 1 to 9 per 100,000 affected people.14 Although the reason for these differences remain unknown, different applied diagnostic criteria could be potential explanations.14 CIDP creates a major burden on patients, from physical to psychosocial aspects, including impaired physical function, pain, fatigue and depression.16 Beyond this, CIDP is associated with significant economic impact due to direct costs of illness and indirect costs of loss of productivity.16

Treatment of patients with CIDP is complex and requires individualised strategies.14 Based on the extensive practical experience of effectiveness, immunomodulation with IVIg or treatment with corticosteroids are effective and strongly recommended by international guidelines for both induction and maintenance treatment.15 Plasma exchange is also an effective treatment option and strongly recommended if IVIg and corticosteroids are ineffective.15

References

1. McCusker C, et al. Allergy Asthma Clin Immunol 2018;14:61;

2. Tangye SG, et al. J Clin Immunol 2022;42:1473–1507;

3. Krivan G, et al. Am J Clin Exp Immunol 2017;6:76–83; I

4. IPOPI. What are Primary Immunodeficiencies (PIDs)? Available at: https://ipopi.org/pids/what-are-pids/; accessed July 2023;

5. Bousfiha A, et al. J Clin Immunol 2020;40:66–81;

6. Modell V, et al. Immunol Res 2011;51:61–70;

7. Wood P. Ther Clin Risk Manag 2012;

8. Espanol T, et al. Patient Prefer Adherence 2014;

9. Bussel J, et al. Expert Rev Hematol 2021;14:1013–1025;

10. NORD. Immune Thrombocytopenia. Available at: https://rarediseases.org/rare-diseases/immune-thrombocytopenia/#affected; accessed July 2023;

11. Sestøl HG, et al. Expert Rev Hematol 2018;11:975–985;

12. Provan D, et al. Blood Adv 2019;3:3780–3817.

13. Briani C, et al. Neurol Sci 2022;43(Suppl 2):605–614;

14. Lehmann HC, et al. J Neurol Neurosurg Psychiatry 2019;90:981–987;

15. Van den Bergh PYK, et al. J Peripher Nerv Syst 2021;26:242–268;

16. Querol L, et al. J Neurol 2021;268:3706–3716.
    

This is an international website for Panzyga® and is intended for healthcare professionals outside the US. The information on this site is not country-specific and may contain information that is outside the approved indications in the country in which you are located.
IMPORTANT: The information on this website is based on the European Summary of Product Characteristics (EU SmPC).
If you wish to contact Octapharma please use the contact form on our corporate website www.octapharma.com.