Experience Sharing of the Latest Approved PK Software from Other Stones (5): British Experience

Recently, the hemophilia A management tool under the guidance of pharmacokinetics (PK) (recombinant human coagulation factor VIII dose calculation software for injection, hereinafter referred to as "PK software") has been approved by the National Medical Products Administration (NMPA) and is applicable to Patients with hemophilia A who are 16 years and older (weight 45kg and above) receiving rAHF-PFM treatment
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Wonderful uninterrupted, uninterrupted knowledge enjoyment
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In this issue, we bring to you the experience of using PK software in the UK, and "adding bricks and tiles" to the experience of using PK software, let's take a look! British experience sharing-PK tool clinical use background preventive treatment is the standard treatment for patients with severe hemophilia A1
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Due to differences in the bleeding phenotypes of patients, under standard-dose preventive treatment, only maintaining the trough FVIII concentration >1 IU/dL will still face the risk of bleeding1
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Therefore, individualized preventive treatment is required to adapt to the patient's bleeding phenotype and life>
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PK-guided individualized preventive treatment can reduce the risk of bleeding in patients with hemophilia, reduce the frequency and dose of coagulation factor infusion, increase factor trough concentration, and reduce medical costs, so that patients are expected to live an ideal life
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However, the traditional detection and calculation of PK parameters are very cumbersome and have a long waiting time, and cannot be widely used in practical applications2
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The approval of PK software makes up for the shortcomings of traditional PK testing
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British experience sharing-PK software clinical use study design 1 A prospective multi-center study, a total of 59 patients from 16 centers were included, of which 16 patients withdrew from the study, and 8 patients did not undergo PK testing
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Use PK software to model the patient's PK curve, and estimate the FVIII trough concentration of the preventive treatment plan (change during treatment) before and after the aid of the PK software
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The patients were grouped according to the trough concentration and bleeding rate at the beginning, middle and end of the study
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British experience sharing-outcome of clinical use of PK software 1 PK software-assisted preventive treatment of bleeding reduced compared with PK software-assisted, after individualized preventive treatment assisted by PK software, the proportion of patients with zero bleeding increased from 36% to 50% , As shown in Figure 1
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Figure 1 Proportion of patients with 0 bleeding before and after PK software assistance.
At the 12th month, the total median bleeding rate decreased (from 1 to 0.
5, P=0.
044) (Figure 2), and the weekly median factor dosage Similar, 86 IU/kg and 82 IU/kg respectively
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Figure 2 Bleeding before PK software assistance vs.
PK software assisted preventive treatment of patients with PK software assisted FVIII trough concentration rises.
Of the patients whose preventive treatment plan was adjusted with the aid of PK software, 13 cases (81%) trough concentration Higher (median> 2 IU/dL), lower bleeding rate (median bleeding rate from 2 to 1)
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Among the patients who adjusted the preventive treatment plan with the aid of PK software, 15 patients (94%) increased the frequency of treatment, and 5 patients received daily treatment for at least a specific time period of a week
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With the aid of PK software, the proportion of patients with the best preventive treatment plan at 12 months increased from 33% to 44% (ie trough concentration> 1, bleeding 0-1), and the proportion of patients with trough concentration <1 decreased from 34% To 23%, the proportion of patients with trough concentration> 1 increased from 66% to 77%
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British experience sharing-patient self-reported PK software use outcome 1 Through the online Haemtrack system, patients self-record treatment and bleeding, 52/54 patients entered the trial 6 months before the trial was enrolled and at the 12th month after the trial started.
Data
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At the beginning of the study, the data of 21 patients (40%) met the data entry standards of the Haemtrack system.
At the middle and end of the study, the data of 40 patients (76%) and 35 (67%) patients met the entry standards
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According to the self-reported data of patients, the dosage of the median factor remained unchanged, and the number of bleeding was reduced after the preventive treatment assisted by PK software (median bleeding from 4 to 2 at 6 months), as shown in Table 1.
: Table 1 Summary of preventive treatment and bleeding of patients who meet the Haemtrack system data entry standards in the trial.
It is known from the experience in the United Kingdom that the assistance of PK software has increased the proportion of patients with FVIII trough concentration and zero bleeding
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PK-guided preventive treatment can develop individualized preventive treatment plans for patients
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Group PK-guided preventive treatment is an important step in individualized treatment
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References: 1.
Saket Badle,et al.
UKPK trial interim analysis: Use of Pharmacokinetic intervention to optimise Factor VIII prophylaxis in severe haemophilia A.
WFH 2018.
2.
https:// Approval number: VV- MEDMAT-55300 Approval time: 10/22/2021 Expiration time: 10/22/2023 Statement This information is intended to help medical and health professionals better understand the latest developments in the field of related diseases
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