Autoimmune diseases
Monday, April 02, 2012, 17:30 – 19:00
Autologous haematopoietic stem cell transplantation in aggressive forms of neuromyelitis optica (NMO) and NMO spectrum disorders
Raffaella Greco, Lucia Moiola, Paolo Rossi, Marta Radaelli, Daniela Ungaro, Massimo Bernardi, Consuelo Corti, Jacopo Peccatori, Attilio Bondanza, Maria Teresa Lupo Stanghellini, Giancarlo Comi, Fabio Ciceri (Milan, IT)
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Background. Neuromyelitis Optica (NMO), an inflammatory and demyelinating autoimmune disorder of the central nervous system, previously considered to be a severe variant of Multiple Sclerosis (MS), is characterized by recurrent attacks of optic neuritis (ON) and by longitudinally extensive trasverse myelitis (LETM). More recently NMO has been recognized as a distinct disorder with positivity for anti-aquaporin 4 (AQP4) antibodies, a poorer prognosis and an unsatisfactory response to most treatments currently in use for MS.
Methods. We evaluated the outcome after high dose immunosuppressive chemotherapy with autologous stem cell transplantation (ASCT) of four patients with aggressive forms of NMO, unresponsive to conventional therapies. All patients had a LETM and 1 also a ON. Patients had a median of 36 years, 3 females and 1 male. Previous treatments included high dose steroids, Cyclophosphamide (CTX), Rituximab, Natalizumab, Plasma Exchange (PEX), Azathioprine, Methotrexate. Mobilization was obtained with CTX 4 gr/mq at day 0 followed by granulocyte colony stimulating factor 5mcg/Kg/day from day +2 to stem cell harvest. HSC harvest was not manipulated before cryopreservation. Three patients received a Thiotepa-CTX based conditioning regimen before ASCT, while one patient a BEAM/ATG/Cyclosporin.
Results. Mobilization was successfull in all cases, with a median of 23,23 x10^6 collected and 6,1 x10^6 infused CD34+cells/Kg. Hematopoietic recovery was documented in all patients, with a median of 13,5 days for neutrophil engraftment and 11 days for platelet. All patients developed febrile neutropenia, two a CMV reactivation, without serious adverse events. Two patients experienced a neurological worsening during the aplasia. All patients are alive. Relapses were observed in all patients within 1 year after ASCT, requiring other treatments, included high dose steroids, Rituximab, PEX, Alemtuzumab and for two patients allogeneic stem cell transplantation (Allo-SCT).
Conclusions. ASCT has been unsatisfactory to maintain long term disease remissions and to reduce disability progression in four patients with aggressive NMO spectrum disorders. Autoimmune Diseases Working Party have launched a retrospective survey of ASCT for NMO and NMO spectrum disorders.
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