Fahd Adeeb1, Shakeel Anjum2, Philip Hodnett3, Ahmad Kashif4, Mary Brady5, Siobhan Morrissey5, Joseph Devlin5, Alexander Duncan Fraser2. 1. Rheumatology Department, University Hospital Limerick, Dooradoyle, Limerick, Ireland; Graduate Entry Medical School, University of Limerick, Limerick, Ireland. Electronic address: fahd_adeeb@yahoo.com. 2. Rheumatology Department, University Hospital Limerick, Dooradoyle, Limerick, Ireland; Graduate Entry Medical School, University of Limerick, Limerick, Ireland. 3. Radiology Department, University Hospital Limerick, Limerick, Ireland. 4. Dermatology Department, University Hospital Limerick, Limerick, Ireland. 5. Rheumatology Department, University Hospital Limerick, Dooradoyle, Limerick, Ireland.
Abstract
OBJECTIVES: This case series explores the potential efficacy of Abatacept in patients presenting with morphea subtypes and deep tissue involvement. METHODS: Three patients with established morphea subtypes and deep tissue involvement and with no contraindication to Abatacept were included in this prospective open-label study. The index patient was exceptionally severely affected with a mean Modified Rodnan Skin Score (MRSS) of 38/51. At baseline, whole-body MRI and skin biopsy were performed which confirmed classical deposition of dense fibrous tissue in the appropriate layer of the skin. MRSS was performed independently by three clinicians and VAS scores (10cm) were measured at baseline for Patient Global Disease Activity (PGDA), Patient Global Pain (PGP), Patient Day Pain (PDP), Patient Night Pain (PNP), and Physician Global Disease Activity (PhGDA). Patients 2 and 3 were similarly screened at baseline except for MRI. Patients were commenced on Abatacept as per body weight (10mg/kg) given intravenously with concomitant tapering dose of oral prednisolone. All three were re-assessed at 6 months and the index case was further re-assessed at 18 months. RESULTS: All patients tolerated the Abatacept well and showed dramatic improvement. The index patient's clinical signs and symptoms, whole-body MRI, and mean Modified Rodnan Skin Score improved dramatically from baseline by 37% at 6 months and by 74% at 18 months. There were no clinically significant adverse outcomes noted. CONCLUSION: We present three cases, one with exceptionally severe disease, which demonstrated excellent clinical response to Abatacept. Abatacept is a promising option for the treatment of severe or resistant morphea, especially in those with deep tissue involvement.
OBJECTIVES: This case series explores the potential efficacy of Abatacept in patients presenting with morphea subtypes and deep tissue involvement. METHODS: Three patients with established morphea subtypes and deep tissue involvement and with no contraindication to Abatacept were included in this prospective open-label study. The index patient was exceptionally severely affected with a mean Modified Rodnan Skin Score (MRSS) of 38/51. At baseline, whole-body MRI and skin biopsy were performed which confirmed classical deposition of dense fibrous tissue in the appropriate layer of the skin. MRSS was performed independently by three clinicians and VAS scores (10cm) were measured at baseline for Patient Global Disease Activity (PGDA), Patient Global Pain (PGP), Patient Day Pain (PDP), Patient Night Pain (PNP), and Physician Global Disease Activity (PhGDA). Patients 2 and 3 were similarly screened at baseline except for MRI. Patients were commenced on Abatacept as per body weight (10mg/kg) given intravenously with concomitant tapering dose of oral prednisolone. All three were re-assessed at 6 months and the index case was further re-assessed at 18 months. RESULTS: All patients tolerated the Abatacept well and showed dramatic improvement. The index patient's clinical signs and symptoms, whole-body MRI, and mean Modified Rodnan Skin Score improved dramatically from baseline by 37% at 6 months and by 74% at 18 months. There were no clinically significant adverse outcomes noted. CONCLUSION: We present three cases, one with exceptionally severe disease, which demonstrated excellent clinical response to Abatacept. Abatacept is a promising option for the treatment of severe or resistant morphea, especially in those with deep tissue involvement.
Authors: Tamás Constantin; Ivan Foeldvari; Clare E Pain; Annamária Pálinkás; Peter Höger; Monika Moll; Dana Nemkova; Lisa Weibel; Melinda Laczkovszki; Philip Clements; Kathryn S Torok Journal: Eur J Pediatr Date: 2018-05-04 Impact factor: 3.183