Doctors involved in treating children with antineutrophil cytpolasmic antibody (ANCA)-associated vasculitis (AAV) have revealed the unmet need for evidence-based treatment guidelines that improve the management and outcomes of their patients, a new international survey revealed.
The study, “Clinical practice variation and need for pediatric-specific treatment guidelines among rheumatologists caring for children with ANCA-associated vasculitis: an international clinician survey,” was published in the Paediatric Rheumatology Online Journal.
ANCA-associated vasculitis refers to a group of rare diseases, such as granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA), characterized by inflammatory cell infiltration and the death of blood vessel walls.
Current childhood AAV treatments include high-dose corticosteroids and cyclophosphamide. But such treatments come with significant adverse effects, and doctors need to finely balance the risks of such treatments with the risk of over- or under-treatment.
In adults, this balance has been fine-tuned with the help of clinical trials, relying on scoring tools that rate disease severity, and accurate disease sub-classification. But due to the rarity of AAV in children, clinical trials are difficult, and pediatric AAV management is based on adult data.
“As a first step toward developing treatment guidelines for pediatric AAV, we sought to better understand the diversity of beliefs and practices associated with current care,” the team wrote.
They surveyed 144 physicians from 36 countries who had seen approximately 1,600 children with GPA or MPA. The survey gathered data regarding the physicians’ experience with pediatric AAV, uptake of existing classification criteria, clinical scoring tools, and treatment guidelines, and variation in current treatment guidelines. They also assessed whether doctors felt the need for pediatric treatment protocols.
Among the respondents, only 14% had seen more than 20 children with AAV. Thirty-nine percent had seen between 10 and 20 patients, and 47% fewer than 10. The majority had seen five or fewer pediatric patients with a new diagnosis of AAV within the past five years.
The team found that despite recent international initiatives regarding classification criteria and treatment guidelines specific to children with GPA and MPA, there has been limited uptake of these criteria by physicians.
Indeed, while all physicians involved in the survey agreed that differentiating between GPA and MPA at the diagnosis stage is important for prognosis, 26% reported they did not always differentiate between the two diseases.
On the other hand, 66% of respondents reported using more than one set of criteria to distinguish GPA and MPA.
Most participants reported using formal assessment tools for determining disease severity (59%) and activity (63%), but only 36% assessed disease damage. Only one third assessed disease damage and activity at every visit. The authors believe that the lack of uptake is due to lack of familiarity and knowledge of existing criteria.
A vast majority of physicians (96%) believed in the need for pediatric treatment guidelines, and 58% were interested in being involved in the process. Only 47% felt that modifying recommendations for adult disease was acceptable for creating the pediatric guidelines.
For most respondents (66%), cyclophosphamide was the first treatment choice as an induction therapy, while 31% opted for Rituxan (rituximab). U.S.-based physicians, those with five or more years of experience, and those who had seen more than 10 patients, were more likely to choose Rituximab as an induction therapy.
The most common maintenance treatment was azathioprine, followed by methotrexate, mycophenolate mofetil, and Rituximab. Physicians with more patient experience were three times more likely to administer maintenance therapy for at least two years, than those who had seen fewer than 10 patients.
Despite the lack of uptake of internationally agreed criteria, most of those surveyed agreed there is a requirement for treatment guidelines that are tailored to children with AAV.
The authors summarize the data by saying that “taken together, these data suggest a need for and interest in consensus treatment guidelines for pediatric GPA and MPA.” They then conclude that establishing standard guidelines for classification and treatment “may facilitate clinician engagement and ultimately lead to improved outcomes for children with AAV.”