Anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis describes a group of rare diseases that cause inflammation in small- to medium-sized blood vessels in your body. This inflammation not only reduces blood flow as a result, but can also damage organs and affect how they work. The exact cause of ANCA-associated vasculitis is currently unknown, but it is thought to be a combination of genetic and environmental factors.
Granulomatosis with polyangiitis (GPA)
GPA is generally characterized by inflammation in the blood vessels that happens when immune cells stick together, called a granuloma. It has a wide range of presentations, but often involves inflammation and damage in the lungs, kidneys, sinuses, ears, nose, and throat.
Microscopic polyangiitis (MPA)
MPA shares some similar symptoms with GPA, but the impact of MPA on your kidneys may be more severe. People with MPA may be at particular risk of uncontrolled bleeding in their lungs.
Some terms you may hear when describing these diseases are: severe and active.
“Severe disease” means that you have signs and/or symptoms of ANCA-associated vasculitis that could be organ- or life-threatening.
“Active disease” means that you have signs and/or symptoms of the disease that are new, not getting better, or getting worse.
About 80-90% of patients with ANCA-associated vasculitis have kidney- or other organ-threatening signs and symptoms, which can be considered severe active disease
GPA and MPA can impact many different parts of your body. This diagram can help you understand some of the signs and symptoms of GPA and MPA. For ~50% of people,* even in remission,† vasculitis symptoms are debilitating, so it is important to keep track of signs and symptoms and discuss them with your doctor. Keeping a notebook or journal can help you have an organized conversation with your doctors to take control of your treatment.
Additionally, symptoms can be persistent. While relapse is common with GPA and MPA, it is often associated with significant harm.
If you notice any of these symptoms, it is important to tell your doctor and discuss which treatments are available to help you manage them. Please note these are just some of the signs and symptoms of severe active GPA or MPA. Severe active GPA or MPA can present differently in each patient.
According to an online, self-administered survey of 100 patients with GPA or MPA from July 21-August 25, 2022.
Remission is the lack of GPA or MPA symptoms on or off immunosuppressive therapy.
When managing severe active GPA or MPA, you and your doctor may face challenges with the disease as well as the medications you may be taking to treat it.
It can often feel difficult or overwhelming managing severe active GPA or MPA. It is possible to address these challenges with your doctor to help manage your GPA or MPA.
Click on each of the challenges below to learn more about managing severe active GPA or MPA, and the importance of talking about these with your doctor.
Tap on each of the challenges below to learn more about managing severe active GPA or MPA, and the importance of talking about these with your doctor.
Since they share many symptoms, diagnosing GPA or MPA can be tricky. However, early diagnosis is extremely important to coming up with a treatment plan that can help manage these conditions.
There are a number of ways your doctor will try to confirm a potential GPA or MPA diagnosis:
Depending on how GPA or MPA has impacted your body, you may need to see different types of doctors, such as a rheumatologist or nephrologist, to successfully manage your symptoms.
Once a diagnosis of GPA or MPA is suspected, a tissue sample of the affected area known as a biopsy may be performed to confirm the presence of vasculitis. Treatment should not necessarily be delayed for a biopsy. However, biopsies can be helpful when trying to understand how much your organs may be impacted by GPA or MPA.
The right treatment may make a difference in GPA or MPA
To help control disease activity, your doctor may prescribe immunosuppressants, such as glucocorticoids.
Immunosuppressants
Immunosuppressants are medications that target inflammation by lowering the activity of the body’s immune system and can be used to induce remission as the standard treatment for severe active GPA and MPA.
Glucocorticoids
Glucocorticoids, such as prednisone, are one type of immunosuppressant. While glucocorticoids can be helpful in controlling disease activity, they can also cause serious side effects. This is especially true if used over a long period. To help, doctors are shifting to treatments that can reduce the time a person takes high-dose glucocorticoids.
Treatment options continue to improve for GPA and MPA patients.
“For someone who is newly diagnosed…ask a lot of questions. Fight for yourself because you’ve got to take care of your whole being. Living a healthy life as a rare disease patient encompasses all facets of life.”
– Brandon, person living with GPA
Have an open conversation
Open conversation between you and your doctor is very important because you both may have different expectations of what successful treatment looks like. Be sure to have open communication on any questions or concerns you may have and let them know of any changes in your health that you may be experiencing.
Below are some questions that can help you and your doctor talk about important topics when it comes to your treatment and expectations.
Prepare for your appointment
It is important to be organized and use your time wisely with your doctor. Follow these tips for a successful visit:
“For someone who is newly diagnosed…ask a lot of questions. Fight for yourself because you’ve got to take care of your whole being. Living a healthy life as a rare disease patient encompasses all facets of life.”
– Brandon, person living with GPA
Connecting with people like you can also provide a sense of community and support. The following organizations provide education, support, and resources that may help you throughout your journey:
The Vasculitis Foundation supports, inspires, and empowers people and families living with vasculitis through a wide range of education, research, clinical, and awareness initiatives.
NORD (National Organization for Rare Disorders) is a patient advocacy organization committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.
The American Kidney Fund (AKF) works on behalf of Americans living with and at risk of kidney disease with programs that support people from prevention through transplant and beyond.
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Recognizing the signs and symptoms of severe active GPA or MPA at an early stage can lead to a timely diagnosis. An early diagnosis allows you and your doctor to work together to decide on a plan to help manage your condition.
However, about 1/3 of people experience a significant diagnostic delay of more than 6 months.
The goal of treatment is to achieve and sustain remission. Remission is the lack of GPA or MPA symptoms on or off immunosuppressive therapy. Response to initial treatment for severe active GPA or MPA varies and some people do not achieve remission.
It is critical to keep track of your symptoms and tell your doctor when you feel like they may be coming back. This can help you and your doctor decide on the best way to manage your GPA or MPA to prevent relapse.
Severe active GPA and MPA are what are known as long-term, progressive diseases. This means that while there may be times where it seems like your symptoms are getting worse, there may also be times where symptoms seem to get better as well.
Relapse is when your GPA or MPA symptoms return after being previously controlled. Preventing relapse is key to staying in remission.
Steroids, called glucocorticoids, are commonly used for GPA and MPA treatment, but they can have side effects.
AAV could lead to impacted organ function, especially in the lungs and kidneys.
AAV threatens the kidneys or another organ in about 80% - 90% of cases, which can be considered severe active disease.
“Quality of Life” describes how well you’re doing and feeling day-to-day. If your treatment plan is working for you, it can improve your quality of life.
Only about 1/4 of people are satisfied with their medication’s ability to control symptoms while maintaining a good quality of life.*
*According to an online, self-administered survey of 100 patients with GPA or MPA from July 21-August 25, 2022.
Anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV) is a group of rare diseases that affects small- to medium-sized blood vessels.1,2 Once affected, these blood vessels become inflamed, leading to reduced blood flow and potentially impaired organ function. The exact cause of ANCA-associated vasculitis is currently unknown, but it is believed to be a combination of genetic and environmental factors.1-3
Granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) are the most common types of AAV and manifest as chronic, progressive autoimmune diseases that impact predominantly small-to-medium blood vessels throughout the body.4-6
GPA: granulomatous inflammation affecting blood vessels involving the upper and lower respiratory tracts and the kidneys; other organs can also be affected7,8
MPA: vasculitis often affecting the kidneys, lungs, and skin7
ACTIVE
New, worsening, or persistent clinical signs and/or symptoms attributed to GPA or MPA that are not related to prior damage9
SEVERE
Vasculitis with life- or organ-threatening manifestations9
Approximately 80-90% of patients with ANCA-associated vasculitis present with renal- or other organ-threatening manifestations, which can be considered severe active disease.9,10
Both the humoral pathway and the alternative complement pathway drive inflammatory vascular injury in GPA and MPA.4,5
The interaction between C5a and one of its receptors, C5aR, plays a critical role in amplifying inflammation and vascular injury in GPA and MPA.3-5
Please note these are just some of the signs and symptoms of severe active GPA or MPA. Severe active GPA or MPA can present differently in each patient.
Clinical Assessment
Serology
Biopsy
Opportunities to further care may start with understanding the humoral and complement mediated pathway of GPA and MPA pathophysiology6,16
ClickTap the icons below to learn more about the potential challenges healthcare professionals face when treating severe active GPA and MPA.
To somebody that’s…diagnosed, the first thing I would tell them is ask a lot of questions. Fight for yourself because you’ve got to take care of your whole being.
of patients are satisfied with their medication’s ability to control symptoms while maintaining a good quality of life30,*
of patients, even in remission, vasculitis symptoms are debilitating30,*
According to an online, self-administered survey of 100 patients with GPA or MPA from July 21-August 25, 2022.
BMI = Body Mass Index.
To somebody that’s…diagnosed, the first thing I would tell them is ask a lot of questions. Fight for yourself because you’ve got to take care of your whole being.
Open conversation between a patient and their HCP is crucial when it comes to treating conditions like severe active GPA or MPA, as each may have different expectations of what successful treatment looks like.27,30 Here we’ve provided some useful topics to help you get the most out of these conversations with your patient.
Your patients may have symptoms that impact their ability to communicate effectively with you, including hearing loss.8 Be sure to consider your patient’s abilities when talking with them, such as facing your patient so they can fully understand you and so that you are both able to read each other’s body language.
Watch a national broadcast Amgen® put together around opportunities to further care in AAV, with a focus on GPA and MPA.
Birmingham Vasculitis Activity Score Resource
The Birmingham Vasculitis Activity Score (BVAS) is a validated tool used to measure disease activity in major studies of vasculitis, including ANCA-associated vasculitis. It captures a broad spectrum of clinical manifestations for new, worsening, or persistent disease across 9 organ systems (1 general and 8 tissue-specific).
ANCA101 Education Resource
This resource provides a summary of the ANCA-associated vasculitis disease mechanism, severe active GPA and MPA manifestations, and challenges with management.
The following organizations provide education, support, and resources that may help your patient throughout their treatment journey.
The Vasculitis Foundation supports, inspires, and empowers people and families living with vasculitis through a wide range of education, research, clinical, and awareness initiatives.
NORD (National Organization for Rare Disorders) is a patient advocacy organization committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.
The American Kidney Fund (AKF) works on behalf of Americans living with and at risk of kidney disease, with programs that support people from prevention through transplant and beyond.
References: 1.Tekin Mİ, Özdal MPC. Acta Medica. 2021;52(4):257-263. 2.Yates M, Watts R. Clin Med (Lond). 2017;17(1):60-64. 3. Shochet L, Holdsworth S, Kitching AR. Front Immunol. 2020;11:525. 4. Kitching AR, Anders H-J, Basu N, et al. Nat Rev Dis Primers. 2020;6(1):71. 5. Al-Hussain T, Hussein MH, Conca W, Al Mana H, Akhtar M. Adv Anat Pathol. 2017;24(4):226-234. 6. Jennette JC, Falk RJ, Bacon PA, et al. Arthritis Rheum 2013;65(1):1-11. doi: 10.1002/art.37715 7. Langford C. Cleve Clin J Med. 2012;79(suppl3):S3-15. 8. Supplement to: Jayne DRW, Merkel PA, Schall TJ, Bekker P; ADVOCATE Study Group. N Engl J Med. 2021;384(7):599-609. 9. Chung SA, Langford CA, Maz M, et al. Arthritis Rheumatol. 2021;73(8):1366-1383. 10. Lamprecht P, Kerstein A, Klapa S, et al. Front Immunol. 2018;9:1-10. 11. Jennette JC, Nachman PH. Clin J Am Soc Nephrol. 2017;12(10):1680-1691. 12. Mukhtyar CB. In: Watts RA, Scott DGI, eds. Vasculitis in Clinical Practice. Springer; 2010:13-19. 13. Qasim A, Patel JB. In: StatPearls [Internet]. StatPearls Publishing; 2023. Accessed October 3, 2023. https://www.ncbi.nlm.nigh.bov/books/NBK554372/ 14. Hunter RW, Welsh N, Farrah TE, et al. BMJ. 2020;369:m1070. 15. Pagnoux C. Eur J Rheumatol. 2016;3(3):122-133. 16. Chen M, Jayne DRW, Zhao M-H. Nat Rev Nephrol. 2017;13(6):359-367. 17. Jones RB, Ferraro AJ, Chaudhry AN, et al. Arthritis Rheum. 2009;60(7):2156-2168. 18. Winkelstein A. Blood. 1973;41(2):273-284. 19. Eickenberg S, Mickholz E, Jung E, et al. Arthritis Res Ther. 2012;14(3):R110. 20. Ogino MH, Prasanna T. In: StatPearls [Internet]. StatPearls Publishing; 2023. Accessed October 3, 2023. https://www.ncbi.nlm.nigh.bov/books/NBK5553087/ 21. Mohammadi O, Kassim TA. In: StatPearls [Internet]. StatPearls Publishing; 2023. 22. Stone JH, Merkel PA, Spiera R, et al; RAVE-ITN Research Group. N Engl J Med. 2010;363(3):221-232. 23. Terrier B, Pagnoux C, Perrodeau E, et al; French Vasculitis Study Group. Ann Rheum Dis. 2018;77(8):1151-1157. 24. Stone JH, McDowell PJ, Jayne DRW, et al. Semin Arthritis Rheum. 2022;55:152010 25. King C, Harper L. Current Treatment Options in Rheumatology. 2017;3(4):230-243. doi.org/10.1007/s40674-017-0082-y 26. Robson J, Doll H, Suppiah R, et al. Rheumatology (Oxford). 2015;54(3):471-481. 27. Robson JC, Dawson J, Cronholm PF, et al. Patient Relat Outcome Meas. 2018;9:17-34. 28. Jayne DRW, Merkel PA, Schall TJ, Bekker P; ADVOCATE Study Group. N Engl J Med. 2021;384(7):599-609. 29. Neumann I. Rheumatology (Oxford). 2020;59(suppl 3):iii60-iii67. 30. Data on file, Amgen; Harris Poll [91973]; 2022. 31. Furuta S, Nakagomi D, Kobayashi Y, et al. JAMA. 2021;325(21):2178-2187.
About one-third of patients experience a significant diagnostic delay of more than 6 months1
Though many patients show renal disease, presenting manifestations can stretch across multiple organ systems2
Presence of common comorbidities (>6%) may further complicate diagnosis3
As a result, 16% of patients have had their referral symptoms for approximately 3 months before receiving their diagnosis3
ENT = ear, nose, throat.; COPD = chronic obstructive pulmonary disease.
COPD = chronic obstructive pulmonary disease; ENT = ear, nose, throat.
Response to induction therapy is variable and many patients do not achieve full clinical remission (defined as a BVAS score of 0 with the patient off glucocorticoids)1
RAVE Clinical Trial: Patients Achieving Full Clinical Remission at 6 Months1
Received one to three pulses of methylprednisolone (1000 mg each) followed by prednisone 1 mg/kg/day.
BVAS = Birmingham Vasculitis Activity Score;
CYC = cyclophosphamide; Q1W = weekly; RTX =
rituximab.
MAINRITSAN-11,2
N = 115 patients with GPA or MPA in complete remission (BVAS = 0) after combined glucocorticoids and “pulse” intravenous CYC
AZA arm (n = 58) received:
2 mg/kg/day until month 12 1.5 mg/kg/day until month 18 1 mg/kg/day until month 22
RTX arm (n = 57) received:
500 mg on days 0 and 14, then months 6, 12, and 18
Prednisone treatment:
Major relapse: reappearance or worsening of disease with BVAS >0 and involvement of at least one major organ, a life-threatening manifestation, or both.
Minor relapse: reappearance or worsening of disease with BVAS >0, not corresponding to a major relapse but requiring mild treatment intensification.
MAINRITSAN-1 Relapse Rates1
AZA = azathioprine; RTX = rituximab. Prednisone dose tapering and the decision to stop prednisone after month 18 were left to each site investigator’s discretion. AZA = azathioprine; BVAS = Birmingham Vasculitis Activity Score; CYC = cyclophosphamide; RTX = rituximab.
Even at lower doses, glucocorticoids can be associated with substantial morbidity1-3
Increased Infections2
Patients receiving glucocorticoid therapy for >6 months have a significantly greater incidence of infections (vs patients not using glucocorticoids after 6 months)*
Patients (N = 147) enrolled in the ANCA disease registry of the GDCN, with an initial diagnosis between 2000 and 2009, and remission on or off therapy attained for at least 1 month.
BMI = body mass index; GDCN = Glomerular Disease Collaborative Network.
Glucocorticoid Toxicity Index (GTI) Assesses Glucocorticoid Toxicity That Can Occur Across Multiple Organ Systems Over Time4
BMI = body mass index.
Renal impairment is common with many patients1,2
GFR = glomerular filtration rate; LTFU = long-term follow-up.
QoL in Patients With AAV vs the General Population2,3
AAV = ANCA-associated vasculitis; QoL = quality of life.
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