Improved Health-related Quality of Life and Physical Function in Patients with Refractory Chronic Gout Following Treatment with Pegloticase: Evidence from Phase III Randomized Controlled Trials

Strand, Vibeke; Khanna, Dinesh; Singh, Jasvinder A.; Forsythe, Anna; Edwards, N. Lawrence

doi:10.3899/jrheum.111375

Cited by 71 publications

(52 citation statements)

References 25 publications

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“…Furthermore, RCTs of pegloticase, a parenteral agent which promotes profound serum urate lowering and rapid clearance of MSU crystals, clearly demonstrate that intensive lowering of serum urate levels leads to reductions in flares and tophus size and to improvements in health-related quality of life and activity limitation, even over a 6-month treatment period 42,43 . In addition, those patients with a durable response to pegloticase (with serum urate level <6 mg/dl) have an enduring improvement in all relevant clinical outcomes 29,30 .…”

Section: Clinical Benefits Of Successful Ultmentioning

confidence: 99%

Discordant American College of Physicians and international rheumatology guidelines for gout management: consensus statement of the Gout, Hyperuricemia and Crystal-Associated Disease Network (G-CAN)

et al. 2017

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Gout is a common form of inflammatory arthritis for which highly effective treatments are available, yet the management of this disease is frequently suboptimal. Gout often presents as an acute 'flare' of painful inflammation that typically resolves within a week or two, but can be difficult to control in some instances, can negatively affect quality of life, can be costly to experience and treat, and can warrant hospital admission. If gout-associated hyperuricaemia is inadequately treated, gout flares often progress in frequency and severity, and a state of chronic, inflammatory arthritis can supervene, leading to continuous pain, decreased joint function, and permanent joint damage.Guidelines for the management of gout are intended to help physicians select the most effective course of treatment and to educate patients in order to ensure adherence. In November 2016, the American College of Physicians (ACP) published a clinical practice guideline for the management of acute and recurrent gout 1 . Despite evaluating similar evidence, the ACP clinical practice guideline differs substantially from all other gout management guidelines issued by major international rheumatology groups in the past 5 years, including the 2012 Abstract | In November 2016, the American College of Physicians (ACP) published a clinical practice guideline on the management of acute and recurrent gout. This guideline differs substantially from the latest guidelines generated by the American College of Rheumatology (ACR), European League Against Rheumatism (EULAR) and 3e (Evidence, Expertise, Exchange) Initiative, despite reviewing largely the same body of evidence. The Gout, Hyperuricemia and Crystal-Associated Disease Network (G-CAN) convened an expert panel to review the methodology and conclusions of these four sets of guidelines and examine possible reasons for discordance between them. The G-CAN position, presented here, is that the fundamental pathophysiological knowledge underlying gout care, and evidence from clinical experience and clinical trials, supports a treat-to-target approach for gout aimed at lowering serum urate levels to below the saturation threshold at which monosodium urate crystals form. This practice, which is truly evidence-based and promotes the steady reduction in tissue urate crystal deposits, is promoted by the ACR, EULAR and 3e Initiative recommendations. By contrast, the ACP does not provide a clear recommendation for urate-lowering therapy (ULT) for patients with frequent, recurrent flares or those with tophi, nor does it recommend monitoring serum urate levels of patients prescribed ULT. Results from emerging clinical trials that have gout symptoms as the primary end point are expected to resolve this debate for all clinicians in the near term future. NATURE REVIEWS | RHEUMATOLOGY VOLUME 13 | SEPTEMBER 2017 | 561 CONSENSUS STATEMENT © 2 0 1 7 M a c m i l l a n P u b l i s h e r s L i m i t e d , p a r t o f S p r i n g e r N a t u r e . A l l r i g h t s r e s e r v e d . UricaseAn enzyme that degrades ur...

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Section: Clinical Benefits Of Successful Ultmentioning

confidence: 99%

Discordant American College of Physicians and international rheumatology guidelines for gout management: consensus statement of the Gout, Hyperuricemia and Crystal-Associated Disease Network (G-CAN)

et al. 2017

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“…Successful reduction of SUA levels by upward titration of ULT, but without prescheduled therapeutic adaptation processes, has been demonstrated in many other trials 38–40 43 44. Patients with gout exhibited a higher velocity of tophus size reduction when reaching a serum urate concentration below the saturation point of MSU early and maintaining that level 39 45 46…”

Section: Resultsmentioning

confidence: 98%

Treat-to-target (T2T) recommendations for gout

et al. 2016

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ObjectivesThe treat-to-target (T2T) concept has been applied successfully in several inflammatory rheumatic diseases. Gout is a chronic disease with a high burden of pain and inflammation. Because the pathogenesis of gout is strongly related to serum urate levels, gout may be an ideal disease in which to apply a T2T approach. Our aim was to develop international T2T recommendations for patients with gout.MethodsA committee of experts with experience in gout agreed upon potential targets and outcomes, which was the basis for the systematic literature search. Eleven rheumatologists, one cardiologist, one nephrologist, one general practitioner and one patient met in October 2015 to develop T2T recommendations based on the available scientific evidence. Levels of evidence, strength of recommendations and levels of agreement were derived.ResultsAlthough no randomised trial was identified in which a comparison with standard treatment or an evaluation of a T2T approach had been performed in patients with gout, indirect evidence was provided to focus on targets such as normalisation of serum urate levels. The expert group developed four overarching principles and nine T2T recommendations. They considered dissolution of crystals and prevention of flares to be fundamental; patient education, ensuring adherence to medications and monitoring of serum urate levels were also considered to be of major importance.ConclusionsThis is the first application of the T2T approach developed for gout. Since no publication reports a trial comparing treatment strategies for gout, highly credible overarching principles and level D expert recommendations were created and agreed upon.

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“…In this issue of The Journal, Strand, et al extend the previous results of these phase-3 studies by presenting the efficacy of pegloticase for patient-reported outcomes (PRO) 13 , in particular the Medical Outcomes Study Short Form-36 (SF-36), which assesses health-related quality of life (HRQOL) in 9 domains, and the Health Assessment Question naire Disability Index (HAQ-DI), which assesses disease-related physical function.…”

mentioning

confidence: 79%

“…Nevertheless, statistically significant improvement in these PRO at the group level does not imply that these differences are meaningful to individuals. Therefore, the use of the minimum clinically important difference (MCID) -the smallest difference in a score that is considered to be worthwhile or important -as in the study by Strand and colleagues 13 , gives an idea of the proportion of patients who feel better. For practical relevance, the proportion of patients receiving pegloticase who reached the Patient Acceptable Symptom State -a state in which patients consider their condition satisfactory or acceptable, and often interpreted as feeling good -would also have been of interest.…”

Section: Rheumatologymentioning

confidence: 99%