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Published on: March 17, 2020
Cyclophosphamide for salvage therapy of chronic graft-versus-host disease: a retrospective analysis
Matthias A Fante1, Barbara Holler2, Daniela Weber2
1Department of Hematology and Oncology, Internal Medicine III, University Hospital Regensburg, Franz-Josef-Strauß-Allee 11, 93053, Regensburg, Germany. matthias.fante@ukr.de.
Insights
Cyclophosphamide (cyclo) shows efficacy as a salvage therapy for chronic graft-versus-host disease (cGvHD), particularly in cGvHD-associated nephritis. This immunosuppressive treatment was well-tolerated in heavily pretreated patients.
Area of Science:
- Hematology
- Immunology
- Nephrology
Background:
- Chronic graft-versus-host disease (cGvHD) is a significant complication following allogeneic stem cell transplantation.
- Steroid-refractory cGvHD and its organ-specific manifestations, such as cGvHD-associated nephritis, present therapeutic challenges.
- Salvage therapy options for advanced or refractory cGvHD are limited, necessitating evaluation of alternative agents.
Purpose of the Study:
- To retrospectively analyze the safety and efficacy of cyclophosphamide (cyclo) as a salvage treatment for chronic graft-versus-host disease (cGvHD).
- To specifically assess cyclo's effectiveness in treating cGvHD-associated (glomerulo-)nephritis and other severe manifestations.
- To evaluate treatment response, tolerability, and complications associated with cyclophosphamide in a heavily pretreated patient cohort.
Main Methods:
- Retrospective analysis of 13 patients treated with cyclophosphamide for moderate to severe steroid-refractory cGvHD between 2010 and 2019.
- Assessment of National Institute of Health organ grading and immunosuppression intensity at baseline and follow-up points (3, 6, 12 months).
- Response evaluation included complete response (CR), partial response (PR), stable disease (SD), minor response (MR), and progressive disease (PD), with overall response rate (ORR) calculated.
Main Results:
- Cyclophosphamide demonstrated an overall response rate (ORR) of 54% (7/13 patients achieving CR or PR).
- Significant and durable responses were observed in all three patients with cGvHD-associated (glomerulo-)nephritis.
- Cyclophosphamide was relatively well-tolerated, with infectious complications >CTCAE grade III in 3/12 patients; no recurrence of underlying malignancy was noted.
Conclusions:
- Cyclophosphamide serves as a potentially effective salvage therapy for refractory chronic graft-versus-host disease, including severe organ manifestations like nephritis.
- The agent was relatively well-tolerated in this cohort of heavily pretreated patients, offering a viable treatment option.
- Further evaluation of cyclophosphamide in prospective clinical trials is warranted to confirm its role in cGvHD management.
Abstract:
We retrospectively analyzed the safety and efficacy of cyclophosphamide (cyclo) for salvage treatment of chronic graft-versus-host disease (cGvHD) and cGvHD-associated (glomerulo-)nephritis at our center between 01/2010 and 11/2019. We identified 13 patients (pts) receiving cyclo for treatment of moderate (3/13) and severe (6/13) steroid-refractory cGvHD, cGvHD-associated (glomerulo-)nephritis (3/13), or vasculitis-like CNS manifestation of cGvHD (1/13). Cyclo was started on median day 509 (range 42-8193) after cGvHD onset; the median duration of application was 153 days (range 14-486) with 2/13 currently continuing treatment. The National Institute of Health organ grading and the intensity of immunosuppression (IS) were assessed at cyclo start and repeated after 3, 6, and 12 months. Response assessment was stopped at the start of any additional new IS. The median time of follow up was 407 days (range 86-1534). Best response was 1/13 CR, 6/13 PR, 4/13 SD, 1/13 MR, and 1/13 PD (ORR 54%). Significant and durable response was observed especially in cGvHD-associated (glomerulo-)nephritis (3/3). Infectious complications > CTCAE grade III were observed in 3/12 pts. During cyclo therapy, none of the pts suffered from recurrence of underlying malignancy. Overall, cyclo was relatively well tolerated and showed responses in heavily pretreated patients but requires further evaluation within clinical trials.
