Panzyga® for your patients with ITP

The efficacy and tolerability of panzyga® in adults with primary chronic immune thrombocytopenia (ITP) were assessed in the NGAM-02 trial (NCT01349790).

ITP is an autoimmune disorder characterised by a low platelet count (<100 × 109/L) with an increased risk of bleeding.1 Although most patients produce antibodies to specific platelet membrane glycoproteins, the trigger for production of autoantibodies against platelets is currently unknown.1 Data on the incidence and prevalence of ITP are scarce.1 The National Organisation of Rare Diseases (NORD) estimates that affected people amount to over 200,000 people worldwide.2 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.3 Patient concerns usually include physical symptoms, social limitations due to the unpredictability of episodes and fear of bleeding in public, psychological effects and fatigue.3 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.1

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 tolerated1 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.1,4

Trial overview5

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*Threshold platelet count <100 × 109/L; other causes of thrombocytopenia excluded through history, physical examination and blood test results.

†Safety, bleeding severity and response rates as defined by the European Medicines Agency guideline on the clinical investigation of human normal immunoglobulin for intravenous administration (IVIg) EMA/CHMP/BPWP/94033/2007 rev. 2) 2010: Alternative response (AR), increase in platelet count to ≥30 x 109/L and to ≥2x baseline platelet count, confirmed on ≥2 separate occasions at least 7 days apart, and absence of bleeding; Complete response (CR), increase in platelet count to ≥100 x 109/L, confirmed on ≥2 separate occasions at least 7 days apart, and absence of bleeding; Loss of AR/CR, AR/CR which subsequently deteriorated, to a platelet count of <30 x 109/L/100 x 109/L or to a level <2x the baseline count, or because of bleeding; No response, Platelet count of <30 x 109/L or <2x increase in baseline platelet count, confirmed on 2 separate occasions ~1 day apart, or the presence of bleeding.

Efficacy

Rapid and sustained high response rates5

Full analysis set (N=36)

Highly significant ~5-fold increase in platelet count was observed mostly within 24 h after the first dose (p<0.0001)5

Normal range platelet counts were observed within 3 days after the first dose5

Platelet count over time in the full analysis set (N =36).

Reduced severity of bleeding5

(Data for 2 patients missing at Day 8)

For almost 80% (18/23) of patients who had bleeding at baseline, the haemorrhages resolved completely by Day 85

Severity of bleeding was assessed by the investigator using a 6-point verbal rating scale: 1=none (no haemorrhage of any kind); 2=minor [few petechiae (≤100 total) and/or ≤5 small bruises (≤3 cm diameter) but no mucosal bleeding]; 3=mild [more than 100 petechiae and/or >5 large bruises (>3 cm diameter) but no mucosal bleeding]; 4=moderate (overt mucosal bleeding, such as epistaxis, gum bleeding, oropharyngeal blood blisters, menorrhagia or gastrointestinal bleeding, not requiring immediate medical intervention); 5=severe [mucosal bleeding or suspected internal haemorrhage (e.g. in the brain, lung, muscle or joint) requiring immediate medical intervention]; 6=life-threatening/fatal (documented intracranial haemorrhage or life-threatening or fatal haemorrhage at any site).5

Tolerability

Favourable tolerability profile5

Safety analysis set (N=40)

Laboratory tests and vital signs assessment demonstrated no significant safety concerns. In each of the haematology and chemistry parameters, changes to abnormal laboratory values considered clinically significant were reported in three and two patients, respectively. Laboratory abnormalities consistent with haemolysis were found in 6 of 40 patients (15.0%), and 1 patient had clinically evident haemolysis that was reported as a TEAE and considered to probably be related to the study drug. Fluctuations in vital signs were minor and within expected range for patients undergoing infusion. No changes in viral status were assessed during the study. Two patients (5.0%) died during the study. Both deaths were deemed not related to the study drug.5

Panzyga® for your patients

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IVIg, immunoglobulin solution for intravenous infusion.

References

Bussel J, et al. Expert Rev Hematol 2021;14:1013–1025;
NORD. Immune Thrombocytopenia. Available at: https://rarediseases.org/rare-diseases/immune-thrombocytopenia/#affected; accessed July 2023;
Sestøl HG, et al. Expert Rev Hematol 2018;11:975–985;
Provan D, et al. Blood Adv 2019;3:3780–3817.
Arbach O, et al. Transfus Med 2019;29:48–54.

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).
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