Am J Blood Res 2013;3(1):84-90
Original Article
Repeated treatment with high dose cyclophosphamide for severe
autoimmune diseases
Amy E DeZern, Michael J Styler, Daniel B Drachman, Laura K Hummers, Richard J Jones, Robert A Brodsky
Division of Hematologic Malignancies, Department of Medical Oncology, The Johns Hopkins School of Medicine; Division of
Hematolo-gy/Oncology, Department of Medicine, Drexel University College of Medicine; Department of Neurology, The Johns
Hopkins School of Medi-cine; Division of Rheumatology, Department of Medicine, The Johns Hopkins School of Medicine;
Division of Hematology, Department of Medicine, The Johns Hopkins School of Medicine, Baltimore, MD, USA
Received December 5, 2012; Accepted January 3, 2013; Epub January 17, 2013; Published January 25, 2013
Abstract: High dose cyclophosphamide (HiCY) without stem cell rescue has been shown to induce remissions in patients
with severe autoimmune disorders (SADS). However, up to 80% of these patients ultimately relapse. Here we review the
outcomes of seven patients treated with two cycles and one patient treated with three cycles of HiCY. The diseases re-treated
were scleroderma, multiple sclerosis, three patients with severe aplastic anemia (SAA), and three patients with myasthenia
gravis (MG). All but two patients with SAA had received standard immunomodulatory therapy for their disease up front and had
been refractory. All patients had complete hematologic recovery. Overall survival in this cohort was 100%. All patients relapsed
after the initial cycle but event free survival thereafter was 93.3%. All are still in remission except two MG patients, one of whom
relapsed after a severe GI infection requiring hospitalization, and the other relapsed 3 years after the second treatment and
she did not respond to the third treatment. This shows that HiCY can be safely re-administered in patients with SAA and
refractory SADS. The quality and duration of second remissions appears to be equal or superior to the first remission.
(AJBR1212002).
Keywords: Autoimmunity, cyclophosphamide, severe autoimmune diseases
Address all correspondence to:
Dr. Amy E DeZern
The Bunting and Blaustein Cancer Research Building I
1650 Orleans Street, Room 3M87
Baltimore, MD 21287, USA.
Phone: 410-5027208; Fax: 410-9558202
E-mail: adezern1@jhmi.edu
Original Article
Repeated treatment with high dose cyclophosphamide for severe
autoimmune diseases
Amy E DeZern, Michael J Styler, Daniel B Drachman, Laura K Hummers, Richard J Jones, Robert A Brodsky
Division of Hematologic Malignancies, Department of Medical Oncology, The Johns Hopkins School of Medicine; Division of
Hematolo-gy/Oncology, Department of Medicine, Drexel University College of Medicine; Department of Neurology, The Johns
Hopkins School of Medi-cine; Division of Rheumatology, Department of Medicine, The Johns Hopkins School of Medicine;
Division of Hematology, Department of Medicine, The Johns Hopkins School of Medicine, Baltimore, MD, USA
Received December 5, 2012; Accepted January 3, 2013; Epub January 17, 2013; Published January 25, 2013
Abstract: High dose cyclophosphamide (HiCY) without stem cell rescue has been shown to induce remissions in patients
with severe autoimmune disorders (SADS). However, up to 80% of these patients ultimately relapse. Here we review the
outcomes of seven patients treated with two cycles and one patient treated with three cycles of HiCY. The diseases re-treated
were scleroderma, multiple sclerosis, three patients with severe aplastic anemia (SAA), and three patients with myasthenia
gravis (MG). All but two patients with SAA had received standard immunomodulatory therapy for their disease up front and had
been refractory. All patients had complete hematologic recovery. Overall survival in this cohort was 100%. All patients relapsed
after the initial cycle but event free survival thereafter was 93.3%. All are still in remission except two MG patients, one of whom
relapsed after a severe GI infection requiring hospitalization, and the other relapsed 3 years after the second treatment and
she did not respond to the third treatment. This shows that HiCY can be safely re-administered in patients with SAA and
refractory SADS. The quality and duration of second remissions appears to be equal or superior to the first remission.
(AJBR1212002).
Keywords: Autoimmunity, cyclophosphamide, severe autoimmune diseases
Address all correspondence to:
Dr. Amy E DeZern
The Bunting and Blaustein Cancer Research Building I
1650 Orleans Street, Room 3M87
Baltimore, MD 21287, USA.
Phone: 410-5027208; Fax: 410-9558202
E-mail: adezern1@jhmi.edu
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