DDAVP trial in discrepant non‐severe haemophilia A patients

Okoye, Helen Chioma; Nielsen, B.; Lee, K.; Abajas, Yasmina L.; Key, Nigel S.; Rollins‐Raval, Marian A.

doi:10.1111/hae.13485

Cited by 2 publications

(8 citation statements)

References 12 publications

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“…In the study by Okoye et al, two of the five patients presented with discrepant FVIII measurements before desmopressin administration. However, these two patients showed no FVIII assay discrepancies 1 hour after desmopressin 7 . When classifying patients according to desmopressin response measured by the OSA or the CSA in our study, regularly patients were classified differently.…”

Section: Discussionmentioning

confidence: 61%

“…Okoye et al also demonstrated that one patient would be classified otherwise according to assay results 1 hour after desmopressin. However, study inclusion criteria differed significantly from our study as patients all had discrepant FVIII assay results before desmopressin administration, with OSA measurements higher than CSA measurements 7 . In our study, we included patients with and without discrepancies before desmopressin administration and both patients with higher FVIII when measured by the OSA as well as the CSA.…”

Section: Discussionmentioning

confidence: 87%

“…FVIII increase was relatively lower when measured by the OSA 1 hour after desmopressin than measured by the CSA, leading to a decrease in relative differences between the two assays. However, this effect was not seen any longer at 2 and 4 hours after desmopressin infusion 7 . As desmopressin effect is tested in all haemophilia A patients, before it is used for treatment and usually measured by only one assay in clinical settings, these findings warrant further research as results may have major implications for therapeutic management.…”

Section: Introductionmentioning

confidence: 94%

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The one‐stage assay or chromogenic assay to monitor baseline factor VIII levels and desmopressin effect in non‐severe haemophilia A: Superiority or non‐inferiority?

Schütte¹,

Hodes²,

Moort³

et al. 2020

Haemophilia

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Introduction: Diagnosis, treatment monitoring and assessment of desmopressin effect in haemophilia A patients are performed by measurement of factor VIII activity (FVIII). The two assays commonly applied are the one-stage assay and the chromogenic assay. Especially in non-severe haemophilia A, discrepancies between these assays are common. It is still unestablished which assay corresponds best with bleeding phenotype and desmopressin effect. Aim: To correlate FVIII levels measured by the one-stage assay and by the chromogenic assay with bleeding phenotype and, additionally, to compare FVIII assay discrepancies before and after desmopressin administration. Method: Factor VIII was measured in 130 non-severe haemophilia A patients during routine visits to the outpatient clinic and/or during desmopressin testing. FVIII was measured by both the one-stage assay and the chromogenic assay. Discrepancies between assays were defined as at least a twofold difference of FVIII or an absolute FVIII difference between measurements of ≥0.10 IU/mL. Bleeding phenotype was defined as annual number of treated bleedings (adjusted ABR). Results: Hundred and thirty non-severe haemophilia A patients were included. In 31/130 patients, assay results were discrepant. However, FVIII measurements with both assays correlated adequately with adjusted ABR. In addition, in 27/130 patients FVIII measurements at baseline and after desmopressin administration were analysed. In 13/27 patients, all measurements were either equivalent or discrepant when results were compared. In 14/27 patients, this was not the case as both equivalent measurements and discrepant measurements at different time points within one patient were observed. Conclusion: Neither the one-stage assay nor the chromogenic assay is superior in predicting bleeding phenotype. In addition, equivalent or discrepant FVIII results measured before desmopressin do not always predict FVIII assay results after desmopressin administration. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.

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Section: Discussionmentioning

confidence: 61%

Section: Discussionmentioning

confidence: 87%

Section: Introductionmentioning

confidence: 94%

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The one‐stage assay or chromogenic assay to monitor baseline factor VIII levels and desmopressin effect in non‐severe haemophilia A: Superiority or non‐inferiority?

Schütte¹,

Hodes²,

Moort³

et al. 2020

Haemophilia

View full text Add to dashboard Cite

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“…The initial literature search identified 236 unique articles, of which 91 were considered eligible for further screening (Figure 1). Twenty‐seven articles mentioned prophylaxis treatment for people with non‐severe HA, of which 15 were prospective 3,5,11–23 and 12 retrospective studies 9,24–34 (cohort studies, n = 10 3,11,13,24,26–28,32–34 ; observational studies, n = 11 12,15–18,22,23,25,29–31,34 ; cross‐sectional studies, n = 2, 5,9 other study types, n = 4 14,19–21 ). Most of the studies (19 out of 27) provided Level IV evidence (well‐designed case‐controlled or cohort studies).…”

Section: Resultsmentioning

confidence: 99%

“…Among eligible studies, treatment regimens were reported for 631/2769 (22.8%) and 676/2291 (29.5%) with mild or moderate HA, respectively 9,12–22,24–34,50 . A total of 550 of people with moderate HA were treated with factor replacement prophylaxis, although the dose, frequency and duration of the prophylaxis regimen was not always mentioned; the vast majority came from western Europe 3,9,13,16–18,24,50 and Australia 28 .…”

Section: Resultsmentioning

confidence: 99%

Prophylaxis use of clotting factor replacement products in people with non‐severe haemophilia: A review of the literature

et al. 2022

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Introduction: People with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life.Aim: To review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making.Methods: A targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life.Results: Although eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n = 668) reported treatment regimens. A total of 549 people with moderate HA were treated with factor replacement prophylaxis and were from high-income countries. On the contrary, nearly all people with mild HA received desmopressin (n = 599). Details of treatment regimens for women with haemophilia and people with HB were sparse. Three studies provided long-term outcomes for people with moderate haemophilia who received prophylaxis with factor concentrate, supporting early prophylaxis in people with a frequent bleeding phenotype regardless of their endogenous clotting factor level to preserve joint health. Conclusion:There remain large knowledge gaps when considering how to provide optimal treatment for people with non-severe haemophilia. Nonetheless, there is a strong rationale that prophylaxis should be considered early in life according to similar strategies as for severe haemophilia for those with a frequent severe bleeding phenotype. K E Y W O R D Sarthropathy, bleeding, episodic/on-demand treatment, non-severe (mild or moderate) haemophilia A or B, prophylactic FVIII or FIX, women with haemophilia (symptomatic carriers)This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

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DDAVP trial in discrepant non‐severe haemophilia A patients

Cited by 2 publications

References 12 publications

The one‐stage assay or chromogenic assay to monitor baseline factor VIII levels and desmopressin effect in non‐severe haemophilia A: Superiority or non‐inferiority?

The one‐stage assay or chromogenic assay to monitor baseline factor VIII levels and desmopressin effect in non‐severe haemophilia A: Superiority or non‐inferiority?

Prophylaxis use of clotting factor replacement products in people with non‐severe haemophilia: A review of the literature

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