Identifying Epidermolysis Bullosa Patient Needs and Perceived Treatment Benefits: An Explorative Study Using the Patient Benefit Index

Schräder, Nicholas H. B.; Korte, Eva W. H.; Duipmans, José C.; Stewart, Roy E.; Bolling, Maria C.; Wolff, André

doi:10.3390/jcm10245836

Cited by 12 publications

(5 citation statements)

References 65 publications

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“…Itch frequency in our cohort was high with occurrence in the preceding month in 227 (93%) of 243 reviews from 50 participants. This concurs with previous studies that demonstrated frequent self-reported itch in 85% (n = 83), [ 10 ] 100% (n = 6) [ 14 , 28 ] and 100% (n = 5) [ 11 ] of individuals with RDEB, although these studies did not break down by RDEB subtype [ 11 , 14 , 15 , 28 ].…”

Section: Discussionsupporting

confidence: 92%

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Itch in recessive dystrophic epidermolysis bullosa: findings of PEBLES, a prospective register study

Mellerio

Pillay

Ledwaba-Chapman

et al. 2023

Orphanet J Rare Dis

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Background Itch is common and distressing in epidermolysis bullosa (EB) but has not previously been studied in depth in different recessive dystrophic EB (RDEB) subtypes. Objectives As part of a prospective register study of the natural history of RDEB we explored features of itch, medications used, and correlation with disease severity and quality of life. Methods Fifty individuals with RDEB aged 8 years and above completed the Leuven Itch Scale (LIS) (total 243 reviews over a 7-year period). Data included itch frequency, severity, duration, distress, circumstances, consequences, itch surface area and medications for itch. The iscorEB disease severity score and the validated EB quality of life tool, QOLEB, were compared to LIS domains and analysed by RDEB subtype. Results Itch was frequent, present in the preceding month in 93% of reviews. Itch severity and distress were significantly greater in severe (RDEB-S) and pruriginosa (RDEB-Pru) subtypes compared to intermediate RDEB (RDEB-I). Itch medications were reported in just over half of reviews including emollients, topical corticosteroids and antihistamines; the proportion of participants not using medication despite frequent pruritus suggests limited efficacy. In inversa RDEB (RDEB-Inv) and RDEB-I, LIS domains correlated with iscorEB and QOLEB. In contrast to previous studies, correlations were lacking in RDEB-S suggesting that global disease burden relatively reduces the contribution of itch. Conclusions This comprehensive study of RDEB-associated itch highlights differences between RDEB subtypes, suggests an unmet need for effective treatments and could serve as control data for future clinical trials incorporating itch as an endpoint.

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Section: Discussionsupporting

confidence: 92%

“…Additionally, patient-reported satisfaction with itch medication was modest. As itch is one of the biggest problems and top priorities for EB patients and caregivers, [ 15 , 28 , 31 , 32 ] this suggests that effective itch treatment remains a significant unmet need in EB.…”

Section: Discussionmentioning

confidence: 99%

Itch in recessive dystrophic epidermolysis bullosa: findings of PEBLES, a prospective register study

Mellerio

Pillay

Ledwaba-Chapman

et al. 2023

Orphanet J Rare Dis

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“…Even though RDEB has been recognized to display features of systemic inflammation leading to progressive multi-organ fibrosis [2,3,8], skin wound closure is consistently identified among the most desired outcomes of disease-modifying treatment [25,26]. As recently reported, systemically administered allogeneic ABCB5 + MSCs have emerged as capable of facilitating complete and durable wound closure in patients with RDEB [20].…”

Section: Discussionmentioning

confidence: 99%

Kinetics of Wound Development and Healing Suggests a Skin-Stabilizing Effect of Allogeneic ABCB5+ Mesenchymal Stromal Cell Treatment in Recessive Dystrophic Epidermolysis Bullosa

Niebergall-Roth

Dieter

Daniele

et al. 2023

Cells

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Recessive dystrophic epidermolysis (RDEB) is a rare, inherited, and currently incurable skin blistering disorder characterized by cyclically recurring wounds coexisting with chronic non-healing wounds. In a recent clinical trial, three intravenous infusions of skin-derived ABCB5+ mesenchymal stromal cells (MSCs) to 14 patients with RDEB improved the healing of wounds that were present at baseline. Since in RDEB even minor mechanical forces perpetually provoke the development of new or recurrent wounds, a post-hoc analysis of patient photographs was performed to specifically assess the effects of ABCB5+ MSCs on new or recurrent wounds by evaluating 174 wounds that occurred after baseline. During 12 weeks of systemic treatment with ABCB5+ MSCs, the number of newly occurring wounds declined. When compared to the previously reported healing responses of the wounds present at baseline, the newly occurring wounds healed faster, and a greater portion of healed wounds remained stably closed. These data suggest a previously undescribed skin-stabilizing effect of treatment with ABCB5+ MSCs and support repeated dosing of ABCB5+ MSCs in RDEB to continuously slow the wound development and accelerate the healing of new or recurrent wounds before they become infected or progress to a chronic, difficult-to-heal stage.

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“…We performed a sample size calculation based on data (n = 28) from a recent study in Dutch patients with EB who had pain scores >3. In 28 patients, mean NRS pain scores were 5.76 ± 1.45 [ 34 ].…”

Section: Methods and Analysismentioning

confidence: 99%

The C4EB study—Transvamix (10% THC / 5% CBD) to treat chronic pain in epidermolysis bullosa: A protocol for an explorative randomized, placebo controlled, and double blind intervention crossover study

Schräder

Duipmans²,

Renken³

et al. 2022

PLoS ONE

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Patients with the genetic blistering skin condition epidermolysis bullosa (EB) report severe pain as a consequence of skin and mucous membrane lesions including blisters, wounds, and scars. Adequate symptom alleviation is not often achieved using conventional pharmacologic interventions. Finding novel approaches to pain care in EB is imperative to improve the quality of life of patients living with EB. There are several anecdotal reports on the use of cannabinoid-based medicines (CBMs) by EB patients to reduce the burden of symptoms. However, controlled clinical investigations assessing these reported effects are lacking. As the pain quality “unpleasantness” delineates EB pain, we hypothesize the modulation of affective pain processing in the brain by way of intervention with CBMs comprising the cannabinoids Δ-9-tetrahydrocannabinol and cannabidiol—objectified by functional magnetic resonance imaging (fMRI). The C4EB study is an investigator-initiated, single-centre, randomized, double-blind, placebo-controlled and crossover trial. Adult patients with the diagnosis epidermolysis bullosa, reporting chronic pain will be eligible to participate. Following baseline measurements, participants will be randomized to receive the sublingually administered interventions placebo and Transvamix® in forward or reversed orders, each for two weeks and separated by a washout. The primary outcome is the difference in numeric rating scale pain scores between grouped interventions, using affective descriptors within the Short-form McGill Pain Questionnaire-2. Secondary outcomes include pain self-efficacy, concomitant analgesic medication-use and adverse events. Additionally, fMRI will be employed to assess brain connectivity related to neuroanatomic pain circuits at baseline, placebo and Transvamix® interventions. The study was approved by the ethical committee at the University Medical Center of Groningen in the Netherlands. Results will be submitted for publication in a peer-reviewed journal. Trial registration number: Netherlands Trial Register: NL9347 (Acronym: C4EB).

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Identifying Epidermolysis Bullosa Patient Needs and Perceived Treatment Benefits: An Explorative Study Using the Patient Benefit Index

Cited by 12 publications

References 65 publications

Itch in recessive dystrophic epidermolysis bullosa: findings of PEBLES, a prospective register study

Itch in recessive dystrophic epidermolysis bullosa: findings of PEBLES, a prospective register study

Kinetics of Wound Development and Healing Suggests a Skin-Stabilizing Effect of Allogeneic ABCB5+ Mesenchymal Stromal Cell Treatment in Recessive Dystrophic Epidermolysis Bullosa

The C4EB study—Transvamix (10% THC / 5% CBD) to treat chronic pain in epidermolysis bullosa: A protocol for an explorative randomized, placebo controlled, and double blind intervention crossover study

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