A total of 192 pediatric patients, median age 8.6 years, with high-risk hematological malignancies, underwent haploidentical stem cell transplantation (haplo-HSCT) using post-transplantation cyclophosphamide (PT-Cy), or ex vivo T cell-depleted (TCD) graft platforms, from January 1999 to December 2016 in 10 centers in Spain. Some 41 patients received an unmanipulated graft followed by PT-Cy for graft-vs-host disease (GvHD) prophylaxis. A total of 151 patients were transplanted with CD3-depleted peripheral blood stem cells (PBSCs) by either CD34 + selection, CD3 + CD19 + depletion, TCRαβ + CD19 + depletion or CD45RA + depletion, added to CD34 + selection for GvHD prophylaxis. The PBSCs were the only source in patients
The association between vasculitis and cancer has been widely reviewed in recent decades. The existence of malignancies in patients with vasculitis has been estimated at about 4.5-8%, haematological neoplasms being the most frequently observed. The haematological malignancies most frequently described are lymphoproliferative diseases such as hairy cell leukaemia and lymphomas. On the contrary, the incidence of paraneoplastic vasculitis in patients with myeloma is low; up to now, we have found nine cases reported on this subject. We report the case of a 73-year-old woman who in 1 year showed three outbreaks of acutely painful, purpuric and ulceronecrotic lesions, localized on the lower extremities. Histopathological study demonstrated thrombosis in the arterioles and leucocytoclastic vasculitis. Complementary tests revealed the presence of multiple myeloma.
Objective
Describe the GETH haploidentical stem cell transplantation (haplo‐HSCT) activity in non‐malignant disease (NMDs).
Methods
We retrospectively analyzed data from children with NMDs who underwent haplo‐HSCT.
Results
From January 2001 to December 2016, 26 pediatric patients underwent 31 haplo‐HSCT through ex vivo T cell‐depleted (TCD) graft platforms or post‐transplantation cyclophosphamide (PT‐Cy) at 7 Spanish centers. Five cases employed unmanipulated PT‐Cy haplo‐HSCT, 16 employed highly purified CD34+ cells, and 10 employed ex vivo TCD grafts manipulated either with CD3+CD19+ depletion, TCRαβ+CD19+ selection or naive CD45RA+ T‐cell depletion. Peripheral blood stem cells were the sole source for patients following TCD haplo‐HSCT, and bone marrow was the source for one PT‐Cy haplo‐HSCT. The most common indications for transplantation were primary immunodeficiency disorders (PIDs), severe aplastic anemia, osteopetrosis, and thalassemia. The 1‐year cumulative incidence of graft failure was 27.4%. The 1‐year III‐IV acute graft‐versus‐host disease (GvHD) and 1‐year chronic GvHD rates were 34.6% and 16.7%, respectively. The 2‐year overall survival was 44.9% for PIDs, and the 2‐year graft‐versus‐host disease‐free and relapse‐free survival rate was 37.6% for the other NMDs. The transplantation‐related mortality at day 100 was 30.8%.
Conclusion
Although these results are discouraging, improvements will come if procedures are centralized in centers of expertise.
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