Abstract
Introduction
Several autoimmune disorders such as multiple sclerosis, Stiff man syndrome or chronic inflammatory demyelinating polyneuropathy (CIDP) can benefit from autologous hematopoietic stem cell transplantation (HSCT) due to its immunomodulatory effects at short- and long-term. Here we present the results from the conduction of an outpatient HSCT procedures in 16 patients with CIDP.
Methods
Prospective study of HSCT in persons with CIDP recruited since 2015. Patients with and without previous immunomodulatory therapy were enrolled and a 3-month wash-out period was recquired; previous or current use of walking support was not a contraindication. All patients signed a consent before starting the study and the study protocol was approved by Clínica Ruiz IRB. Before the conditioning, all participants underwent a comprehensive evaluation. On day -11 and -10, patients received cyclophosphamide (Cy) at 50 mg/kg, followed by a 7-day course of G-CSF previous to cell collection (day -2) through apheresis using a Mahurkar catheter. Then, on day -2 and -1 a second round of Cy at 50 mg/kg was given (Figure 1). Transfusions and antimicrobial prophylaxis were administered per local protocol. Upon hematological recovery (>500 ANC) all patients received a rituximab infusion at 1000 mg.
Results
Sixteen patients with CIDP underwent screening for transplantation clearance and all of them proceeded to HSCT. Of those, 8 (50%) patients were male. Median age at transplantation was found to be 44 years (SD 10.5). Walking support was employed at the time of HSCT by 7 (44%) patients and most frequent previous therapy was intravenous immunoglobulin in 4 (25%) patients. On average a mean 8.5x10 6/kg (SD 4.2) of CD34 + cells were infused. The mean nadir neutropenia was found to be 0.11x10 9/L (SD 0.13) and the median time of granulocytes recovery was 7.0 days (SD 2.9) while median time of platelet recovery was 5.5 days (SD 3.0). One (6%) patient was admitted to hospital due to febrile neutropenia and 4 (25%) patients required red-blood cell and platelet transfusion support. In 3 of 5 persons the need of walking support reverted.
Conclusions
Our experience suggests that people with CIDP may undergo HSCT with no major complications on an outpatient basis. Hospital admissions, hematological recovery, and infectious complications seem to be relatively low-severity complications that could be manage with standard protocols.
Gomez-Almaguer: Bristol-Myers-Squibb: Honoraria, Speakers Bureau; Roche: Honoraria, Speakers Bureau; Takeda: Honoraria, Speakers Bureau; Janssen: Honoraria, Speakers Bureau.
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