Evolution, trends, outcomes, and economics of hematopoietic stem cell transplantation in severe autoimmune diseases

Abstract

Key Points Using EBMT data in >2000 patients from 1994 to 2015, we analyzed the development and factors influencing outcomes of HSCT for ADs. Transplant center experience, accreditation, and national socioeconomic factors are relevant for outcomes and future health service delivery.

Incorporation of thiotepa in a reduced intensity conditioning regimen may improve engraftment after transplant for HLH

“…The third patient had underlying CAEBV and developed Grade IV GVHD six months post‐transplant and died from complications of intensive immunosuppression. Of note, all three patients that died had HLH associated with conditions that have been associated with poor outcomes after HSCT, namely STAT3 GOF, systemic JIA and CAEBV (Snowden et al , ; Snowden et al , ; Sawada & Inoue, ; Fabre et al , ).…”

Autologous stem cell transplantation for refractory opsoclonus myoclonus ataxia syndrome

“…Thus, implementing an effective therapy for this syndrome is paramount in order to diminish these long‐term effects. ASCT are increasingly being used to treat autoimmune diseases . Transplants for autoimmune diseases with elements of neuroinflammation such as chronic demyelinating polyneuropathy, multiple sclerosis, and myasthenia gravis has been reported .…”

“…12 Transplants for autoimmune diseases with elements of neuroinflammation such as chronic demyelinating polyneuropathy, multiple sclerosis, and myasthenia gravis has been reported. 12 The rationale for the use of ASCT for autoimmune disease is to remove pathogenic autoreactive cells and replace them with de novo repertoire of immune cells. 13 Both of our patients tolerated transplant well with different responses in the two patients.…”

Parental experiences in end‐of‐life decision‐making in allogeneic pediatric stem cell transplantation

“…In Europe, annually over 5000 pediatric HSCTs are performed, of which 75% are allogeneic 1 . Despite improved HSCT outcomes over the last decades, 2‐7 a significant number of pediatric patients still die after a HSCT because of recurrence of initial disease or HSCT complications. Survival rates ranged widely from 45 to 95% based on the initial disease and transplant modality 2‐7 …”

“…Survival rates ranged widely from 45 to 95% based on the initial disease and transplant modality. [2][3][4][5][6][7] Parents are confronted with their child's somatic and psychosocial distress during HSCT [8][9][10] and they experience intense distress and disorientation themselves. [11][12][13] Their focus during the child's end-oflife (EOL) period includes mainly cure 14 and it has been shown that bereaved parents are at great risk of decreased psychological wellbeing.…”