A randomized, double-blind, placebo-controlled trial of photopheresis in systemic sclerosis

Knobler, Robert; French, Lars E.; Kim, Youn; Bisaccia, Emil; Graninger, Winfried; Nahavandi, Hesam; Strobl, Frank J.; Keystone, Edward; Mehlmauer, Marilyn; Rook, Alain H.; Braverman, Irwin M.

doi:10.1016/j.jaad.2005.11.1091

Cited by 113 publications

(108 citation statements)

References 22 publications

(21 reference statements)

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“…Comparison of skin scores between the two study arms did not achieve statistical significance because of the small sample size of the study arms. Joint involvement was also significantly improved after 6 months and 12 months of active ECP when compared with baseline [13]. Based on the available literature, especially patients with early stage of disease and significant skin involvement seem to respond best to ECP.…”

Section: Systemic Sclerosismentioning

confidence: 85%

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Clinical Results of Extracorporeal Photopheresis

Worel

Leitner²

2012

Transfus Med Hemother

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Extracorporeal photopheresis (ECP) is a combination of leukapheresis and photodynamic therapy in which blood is treated with photoactivable drugs which are then activated with ultraviolet light and re-infused to the patient. It has been used successfully for more than 30 years in the treatment of erythrodermic cutaneous T-cell lymphoma (CTCL) and over 20 years for chronic graft-versus-host disease (GVHD). ECP has also shown promising results in the treatment of acute GVHD and other T-cellmediated diseases, including systemic sclerosis, treatment and prevention of solid organ rejection, and more recently Crohn’s disease. The use of ECP may allow a significant reduction or even discontinuation of corticosteroids and/or other immunosuppressants, thus leading to reduced long-term morbidity and mortality and improved overall survival. ECP is a well-tolerated therapy. No significant side effects have been reported during the last 30 years. It has been shown that ECP is not associated with an increased incidence of infections, malignancies, or recurrence of underlying malignant disease, neither during short-term nor during long-term therapy.

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Section: Systemic Sclerosismentioning

confidence: 85%

“…Subsequently, a randomized, double-blind, placebo-controlled trial conducted at 16 investigational sites in the USA, Canada, and Europe was initiated by Knobler et al [13]. Patients were randomized at baseline to receive either active or sham ECP according to a secure computer-generated randomization schedule.…”

Section: Systemic Sclerosismentioning

confidence: 99%

Clinical Results of Extracorporeal Photopheresis

Worel

Leitner²

2012

Transfus Med Hemother

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“…These findings could not be confirmed by Zachariae et al [75] in a study performed on 8 patients with progressive systemic sclerosis. However, Knobler et al [76] very recently demonstrated the effectiveness of ECP in a 16-center, randomized, doubleblind placebo-controlled trial performed on 64 patients with disease duration < 2 years. The authors observed a significant improvement in skin scores (p = 0.0024) in patients who underwent ECP compared to those who underwent sham apheresis.…”

Section: Miscellaneous Autoimmune Diseasesmentioning

confidence: 99%

Extracorporeal Photochemotherapy as a Challenging Treatment for Cutaneous T-Cell Lymphoma, Acute and Chronic Graft-versus-Host Disease, Organ Rejection and T-Lymphocyte-Mediated Autoimmune Diseases

Perseghin¹

2007

Transfus Med Hemother

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20 years ago, in 1987, Edelson and co-workers published their first report on the effectiveness of a new procedure, called extracorporeal photochemotherapy (ECP), in patients with advanced stage cutaneous T-cell lymphoma (CTCL). The positive response (>70% overall) achieved in those patients encouraged several groups to try out this new technology in other T-lymphocyte-mediated autoimmune diseases and a number of dermatological diseases, which sometimes gave conflicting results. In the following years, ECP obtained FDA approval as first line treatment in CTCL. In the 1990s ECP was applied to acute and chronic graft-versus-host disease (GvHD) refractory to conventional immunosuppressive therapy and proved to be effective in >60% of cases of this larger patient population. Today, although the effectiveness of ECP in GvHD is generally acknowledged, this is mainly based on retrospective or observational studies, as data from large, randomized multicenter trials, has yet to be published. Moreover, ECP’s real mechanism of action and optimal treatment schedule are still under investigation. The aim of this review is to summarize knowledge acquired to date about ECP.

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“…The original process was finalized by Edelson in the 1980s, and preliminary results published in 1987 for the treatment of cutaneous T-cell lymphoma [1] were strong enough to make ECP the first anti-tumoral immunotherapy to gain FDA approval, in 1988 [2]. Subsequently, its supposed anti-clonotypic effect directed against pathogenic T-cell clones and its excellent safety profile have prompted assessments of ECP in a multitude of T-cell-mediated diseases, with sometimes very encouraging but sometimes disappointing results ( Table 1) [3][4][5][6]. Of note, ECP has never been proved to offer any survival advantage in a context of a randomized trial.…”

mentioning

confidence: 99%

“…Placebo-controlled randomized trials assessing ECP [3,4,8,14,15] Table 2. Proposed mechanisms of action of ECP [10].…”

mentioning

confidence: 99%