Dermatomyositis and Polymyositis (DM/PM)

Patel A, Seely G, and Aggarwal R ―Case Reports in Rheumatology. 2016.

Acthar Gel was assessed in DM/PM patients who had active disease despite previous treatment¹

Study Design

Results

STUDY OBJECTIVE

A retrospective case series of 4 DM/PM patients to evaluate the efficacy and safety of Acthar Gel in mediating progression¹

Four patients with DM (n=2) or PM (n=2) were treated with Acthar Gel 80 U twice weekly subcutaneously in conjunction with other therapies
- All patients had received ≥2 previous treatments with incomplete response
- All patients also received prednisone, in doses ranging from 5 to 30 mg/day
- Other concomitant medications allowed during the study included rituximab, azathioprine, intravenous immunoglobulin, methotrexate, and mycophenolate mofetil

Patient Characteristics

Myositis	Mean age
2 patients with DM 2 patients with PM	57.3 years
Previous treatments	Concomitant treatments*
Prednisone Intravenous steroids Mycophenolate mofetil Azathioprine Cyclosporine Etanercept Infliximab Intravenous immunoglobulin Adalimumab Thalidomide Methotrexate Hydroxychloroquine Quinacrine Ustekinumab Rituximab	Prednisone Rituximab Methotrexate Azathioprine Intravenous immunoglobulin Mycophenolate mofetil

Myositis	Mean age	Previous treatments	Concomitant treatments*
2 patients with DM 2 patients with PM	57.3 years	Prednisone Intravenous steroids Mycophenolate mofetil Azathioprine Cyclosporine Etanercept Infliximab Intravenous immunoglobulin Adalimumab Thalidomide Methotrexate Hydroxychloroquine Quinacrine Ustekinumab Rituximab	Prednisone Rituximab Methotrexate Azathioprine Intravenous immunoglobulin Mycophenolate mofetil

*Not every patient was on the same concomitant treatments.

Study Limitations

These results are based on a retrospective 4-patient case series and may not be fully representative of outcomes in the overall patient population. All 4 patients were on multiple therapies. The clinical outcomes may not be solely attributable to Acthar Gel.

All patients showed improvement in muscle strength, as well as resolution of rash¹

Summary Results

3 patients improved after treatment with Acthar Gel; 1 patient did not respond
Acthar Gel did not exacerbate any comorbidity and no significant changes in blood pressure, weight, or glycemic control were observed

Clinical assessments for each case before and after treatment with Acthar Gel

Case: Age/ gender/ diagnosis	CPK (total 24-173)		Muscle weakness^†
	Before	After	Before	After
Case 1: 70/F DM	48	72	None	None
Case 2: 50/M DM	143	151	None	None
Case 3: 52/F PM	1785	132	Mild	None
Case 4: 57/M PM	6753	403	Severe	None
Case: Age/ gender/ diagnosis	Myalgia		Prednisone dose
	Before	After	Before	After
Case 1: 70/F DM	None	None	60 mg	None
Case 2: 50/M DM	Mild	None	30 mg	5 mg
Case 3: 52/F PM	Moderate	Mild	20 mg	5 mg
Case 4: 57/M PM	Severe	None	10 mg	None
Case: Age/ gender/ diagnosis	Rash^‡
	Before		After
Case 1: 70/F DM	Active		Mostly resolved
Case 2: 50/M DM	Active		No worsening
Case 3: 52/F PM	N/A		N/A
Case 4: 57/M PM	N/A		N/A

Case: Age/gender/ diagnosis	CPK (total 24-173)		Muscle weakness^†		Myalgia		Prednisone dose		Rash^‡
	Before	After	Before	After	Before	After	Before	After	Before	After
Case 1: 70/F DM	48	72	None	None	None	None	60 mg	None	Active	Mostly resolved
Case 2: 50/M DM	143	151	None	None	Mild	Mild	30 mg	5 mg	Active	No worsening
Case 3: 52/F PM	1785	132	Mild	None	Moderate	None	20 mg	5 mg	N/A	N/A
Case 4: 57/M PM	6753	403	Severe	None	Severe	None	10 mg	None	N/A	N/A

CPK=creatine phosphokinase.

^†Measured via manual muscle testing and converted to a generalized scale (0, 1, 2=severe; 3=moderate; 4=mild; 5=normal).

^‡In one dermatomyositis patient, rash mostly resolved and a flare-up of the erythematous rash after sun exposure resolved. In the other dermatomyositis patient, rash remained active but did not worsen.

Safety Findings

Acthar Gel did not exacerbate any comorbidity and no significant changes in blood pressure, weight, or glycemic control were observed
- 1 patient had mild weight gain, which could be attributed to concomitant prednisone
No changes in skin pigmentation
1 patient reported blurry vision during Acthar Gel treatment. However, this condition resolved while the patient was still receiving Acthar Gel therapy
Bone density improved in 1 patient, despite exclusion of bisphosphonates

NEXT: Retrospective Case Series (Levine et al 2012)

References

Explore the potential MOA of Acthar Gel

Learn more

Learn about at-home injection training for Acthar Gel patients

Learn more

IMPORTANT SAFETY INFORMATION

Contraindications

Acthar should never be administered intravenously
Administration of live or live attenuated vaccines is contraindicated in patients receiving immunosuppressive doses of Acthar
Acthar is contraindicated where congenital infections are suspected in infants
Acthar is contraindicated in patients with scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of a peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency, adrenocortical hyperfunction, or sensitivity to proteins of porcine origin

INDICATIONS

Acthar^® Gel (repository corticotropin injection) is indicated for:

Treatment during an exacerbation or as maintenance therapy in selected cases of systemic lupus erythematosus
Treatment during an exacerbation or as maintenance therapy in selected cases of dermatomyositis (polymyositis)
Adjunctive therapy for short-term administration (to tide the patient over an acute episode or exacerbation) in: psoriatic arthritis; rheumatoid arthritis, including juvenile rheumatoid arthritis (selected cases may require low-dose maintenance therapy); ankylosing spondylitis
Treatment of symptomatic sarcoidosis

Warnings and Precautions

The adverse effects of Acthar are related primarily to its steroidogenic effects
Acthar may increase susceptibility to new infection or reactivation of latent infections
Suppression of the hypothalamic-pituitary-adrenal (HPA) axis may occur following prolonged therapy with the potential for adrenal insufficiency after withdrawal of the medication. Adrenal insufficiency may be minimized by tapering of the dose when discontinuing treatment. During recovery of the adrenal gland patients should be protected from the stress (e.g. trauma or surgery) by the use of corticosteroids. Monitor patients for effects of HPA suppression after stopping treatment
Cushing’s syndrome may occur during therapy but generally resolves after therapy is stopped. Monitor patients for signs and symptoms
Acthar can cause elevation of blood pressure, salt and water retention, and hypokalemia. Blood pressure, sodium, and potassium levels may need to be monitored
Acthar often acts by masking symptoms of other diseases/disorders. Monitor patients carefully during and for a period following discontinuation of therapy
Acthar can cause GI bleeding and gastric ulcer. There is also an increased risk for perforation in patients with certain gastrointestinal disorders. Monitor for signs of bleeding
Acthar may be associated with central nervous system effects ranging from euphoria, insomnia, irritability, mood swings, personality changes, and severe depression to psychosis. Existing conditions may be aggravated
Patients with comorbid disease may have that disease worsened. Caution should be used when prescribing Acthar in patients with diabetes and myasthenia gravis
Prolonged use of Acthar may produce cataracts, glaucoma, and secondary ocular infections. Monitor for signs and symptoms
Acthar is immunogenic and prolonged administration of Acthar may increase the risk of hypersensitivity reactions. Neutralizing antibodies with chronic administration may lead to loss of endogenous ACTH activity
There is an enhanced effect in patients with hypothyroidism and in those with cirrhosis of the liver
Long-term use may have negative effects on growth and physical development in children. Monitor pediatric patients
Decrease in bone density may occur. Bone density should be monitored for patients on long-term therapy
Pregnancy Class C: Acthar has been shown to have an embryocidal effect and should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus

Adverse Reactions

Common adverse reactions for Acthar are similar to those of corticosteroids and include fluid retention, alteration in glucose tolerance, elevation in blood pressure, behavioral and mood changes, increased appetite, and weight gain
Specific adverse reactions reported in IS clinical trials in infants and children under 2 years of age included: infection, hypertension, irritability, Cushingoid symptoms, constipation, diarrhea, vomiting, pyrexia, weight gain, increased appetite, decreased appetite, nasal congestion, acne, rash, and cardiac hypertrophy. Convulsions were also reported, but these may actually be occurring because some IS patients progress to other forms of seizures and IS sometimes masks other seizures, which become visible once the clinical spasms from IS resolve

Other adverse events reported are included in the full Prescribing Information.

Please see full Prescribing Information for additional Important Safety Information.

Acthar Gel was assessed in DM/PM patients who had active disease despite previous treatment1

STUDY OBJECTIVE

A retrospective case series of 4 DM/PM patients to evaluate the efficacy and safety of Acthar Gel in mediating progression1

Patient Characteristics

Study Limitations

All patients showed improvement in muscle strength, as well as resolution of rash1

Summary Results

Safety Findings

You might also like

IMPORTANT SAFETY INFORMATION

Contraindications

INDICATIONS

IMPORTANT SAFETY INFORMATION

Contraindications

INDICATIONS

Warnings and Precautions

Adverse Reactions

Acthar Gel was assessed in DM/PM patients who had active disease despite previous treatment¹

A retrospective case series of 4 DM/PM patients to evaluate the efficacy and safety of Acthar Gel in mediating progression¹

All patients showed improvement in muscle strength, as well as resolution of rash¹