Abstract
BACKGROUND: Results in murine and nonhuman primate suggested that the bone marrow (BM) might be an alternative site for pancreatic islet transplantation. METHODS: We report the results of 2 clinical studies in patients with type 1 diabetes receiving an intra-BM allogeneic islet transplantation: a feasibility study in patients with hepatic contraindications for liver islet allotransplantation receiving a single intra-BM islet infusion (n = 4) and a pilot randomized trial (1:1 allocation using blocks of size 6) in which patients were randomized to receive islets into either the liver (n = 6) or BM (n = 3) to evaluate islet transplant function and survival. RESULTS: We observed no adverse events related to the intrabone injection procedure or the presence of islets in the BM. None of the recipient of an intra-BM allogeneic islet transplantation had a primary nonfunction, as shown by measurable posttransplantation C-peptide levels and histopathological evidence of insulin-producing cells or molecular markers of endocrine tissue in BM biopsy samples collected during follow-up. All patients receiving islets in the BM except 1 lost islet function during the first 4 months after infusion (2 with an early graft loss). Based on biopsies and immunomonitoring, we concluded that the islet loss was primarily caused by the recurrence of autoimmunity. CONCLUSIONS: Bone marrow is not a suitable alternative site for pancreatic islet allotransplantation in patients with type 1 diabetes.
| Original language | English |
|---|---|
| Pages (from-to) | 839-851 |
| Number of pages | 13 |
| Journal | Transplantation |
| Volume | 103 |
| Issue number | 4 |
| DOIs | |
| Publication status | Published - Apr 1 2019 |
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ASJC Scopus subject areas
- Transplantation
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Islet Allotransplantation in the Bone Marrow of Patients With Type 1 Diabetes : A Pilot Randomized Trial. / Maffi, Paola; Nano, Rita; Monti, Paolo; Melzi, Raffaella; Sordi, Valeria; Mercalli, Alessia; Pellegrini, Silvia; Ponzoni, Maurilio; Peccatori, Jacopo; Messina, Carlo; Nocco, Angela; Cardillo, Massimo; Scavini, Marina; Magistretti, Paola; Doglioni, Claudio; Ciceri, Fabio; Bloem, Stef J.; Roep, Bart O.; Secchi, Antonio; Piemonti, Lorenzo.
In: Transplantation, Vol. 103, No. 4, 01.04.2019, p. 839-851.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Islet Allotransplantation in the Bone Marrow of Patients With Type 1 Diabetes
T2 - A Pilot Randomized Trial
AU - Maffi, Paola
AU - Nano, Rita
AU - Monti, Paolo
AU - Melzi, Raffaella
AU - Sordi, Valeria
AU - Mercalli, Alessia
AU - Pellegrini, Silvia
AU - Ponzoni, Maurilio
AU - Peccatori, Jacopo
AU - Messina, Carlo
AU - Nocco, Angela
AU - Cardillo, Massimo
AU - Scavini, Marina
AU - Magistretti, Paola
AU - Doglioni, Claudio
AU - Ciceri, Fabio
AU - Bloem, Stef J.
AU - Roep, Bart O.
AU - Secchi, Antonio
AU - Piemonti, Lorenzo
PY - 2019/4/1
Y1 - 2019/4/1
N2 - BACKGROUND: Results in murine and nonhuman primate suggested that the bone marrow (BM) might be an alternative site for pancreatic islet transplantation. METHODS: We report the results of 2 clinical studies in patients with type 1 diabetes receiving an intra-BM allogeneic islet transplantation: a feasibility study in patients with hepatic contraindications for liver islet allotransplantation receiving a single intra-BM islet infusion (n = 4) and a pilot randomized trial (1:1 allocation using blocks of size 6) in which patients were randomized to receive islets into either the liver (n = 6) or BM (n = 3) to evaluate islet transplant function and survival. RESULTS: We observed no adverse events related to the intrabone injection procedure or the presence of islets in the BM. None of the recipient of an intra-BM allogeneic islet transplantation had a primary nonfunction, as shown by measurable posttransplantation C-peptide levels and histopathological evidence of insulin-producing cells or molecular markers of endocrine tissue in BM biopsy samples collected during follow-up. All patients receiving islets in the BM except 1 lost islet function during the first 4 months after infusion (2 with an early graft loss). Based on biopsies and immunomonitoring, we concluded that the islet loss was primarily caused by the recurrence of autoimmunity. CONCLUSIONS: Bone marrow is not a suitable alternative site for pancreatic islet allotransplantation in patients with type 1 diabetes.
AB - BACKGROUND: Results in murine and nonhuman primate suggested that the bone marrow (BM) might be an alternative site for pancreatic islet transplantation. METHODS: We report the results of 2 clinical studies in patients with type 1 diabetes receiving an intra-BM allogeneic islet transplantation: a feasibility study in patients with hepatic contraindications for liver islet allotransplantation receiving a single intra-BM islet infusion (n = 4) and a pilot randomized trial (1:1 allocation using blocks of size 6) in which patients were randomized to receive islets into either the liver (n = 6) or BM (n = 3) to evaluate islet transplant function and survival. RESULTS: We observed no adverse events related to the intrabone injection procedure or the presence of islets in the BM. None of the recipient of an intra-BM allogeneic islet transplantation had a primary nonfunction, as shown by measurable posttransplantation C-peptide levels and histopathological evidence of insulin-producing cells or molecular markers of endocrine tissue in BM biopsy samples collected during follow-up. All patients receiving islets in the BM except 1 lost islet function during the first 4 months after infusion (2 with an early graft loss). Based on biopsies and immunomonitoring, we concluded that the islet loss was primarily caused by the recurrence of autoimmunity. CONCLUSIONS: Bone marrow is not a suitable alternative site for pancreatic islet allotransplantation in patients with type 1 diabetes.
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UR - http://www.scopus.com/inward/citedby.url?scp=85063712502&partnerID=8YFLogxK
U2 - 10.1097/TP.0000000000002416
DO - 10.1097/TP.0000000000002416
M3 - Article
C2 - 30130323
AN - SCOPUS:85063712502
VL - 103
SP - 839
EP - 851
JO - Transplantation
JF - Transplantation
SN - 0041-1337
IS - 4
ER -