New perspectives for children with microvillous inclusion disease

Early small bowel transplantation

Frank M. Ruemmele, Dominique M. Jan, Florence Lacaille, Jean Pierre Cézard, Danielle Canioni, Alan D. Phillips, Michel Peuchmaur, Yves Aigrain, Nicole Brousse, Jacques Schmitz, Yann Revillon, Oliver Goulet

Research output: Contribution to journalArticle

50 Citations (Scopus)

Abstract

Background. Microvillous inclusion disease (MVID) is a congenital intestinal epithelial cell disorder leading to lifelong intestinal failure. Despite long-term total parenteral nutrition, life expectancy is extremely reduced because of metabolic or septic complications or liver failure. Methods. Twelve patients with early-onset MVID were evaluated between 1995 and 2002 for the possibility of small bowel transplantation (SbTx). Three patients died before they could be placed on the waiting list for SbTx, and one patient is still awaiting SbTx. SbTx was contraindicated in one patient. Results. Seven of 12 patients (six boys and one girl) underwent transplantation (three SbTxs and four combined liver-SbTxs). Actuarial survival rates were 100% and 75% in the SbTx and combined liver-SbTx groups, respectively, with a mean follow-up of 3 years (1.1-8.5 years). In contrast, the survival rate was only 40% in the subgroup of five patients who did not undergo transplantation. After transplantation, all patients were weaned from parenteral nutrition: the five patients with an additional colon graft were weaned within 36 days as opposed to the others without colonic transplant who obtained full intestinal autonomy several months after transplantation. The only two surviving patients who did not undergo SbTx remain highly dependent on total parenteral nutrition, which is complicated by repeated episodes of metabolic decompensation. Conclusions. SbTx alone or in combination with the liver is highly successful in children with MVID, offering them a long-term perspective for the first time. Associated colon grafting markedly improves the outcome and quality of life after SbTx in patients with MVID.

Original languageEnglish (US)
Pages (from-to)1024-1028
Number of pages5
JournalTransplantation
Volume77
Issue number7
DOIs
StatePublished - Apr 15 2004
Externally publishedYes

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Cytomegalovirus Infections
Transplantation
Total Parenteral Nutrition
Liver
Colon
Survival Rate
Transplants
Waiting Lists
Parenteral Nutrition
Liver Failure
Life Expectancy
Epithelial Cells
Quality of Life

ASJC Scopus subject areas

  • Transplantation
  • Immunology

Cite this

Ruemmele, F. M., Jan, D. M., Lacaille, F., Cézard, J. P., Canioni, D., Phillips, A. D., ... Goulet, O. (2004). New perspectives for children with microvillous inclusion disease: Early small bowel transplantation. Transplantation, 77(7), 1024-1028. https://doi.org/10.1097/01.TP.0000119163.30745.C1

New perspectives for children with microvillous inclusion disease : Early small bowel transplantation. / Ruemmele, Frank M.; Jan, Dominique M.; Lacaille, Florence; Cézard, Jean Pierre; Canioni, Danielle; Phillips, Alan D.; Peuchmaur, Michel; Aigrain, Yves; Brousse, Nicole; Schmitz, Jacques; Revillon, Yann; Goulet, Oliver.

In: Transplantation, Vol. 77, No. 7, 15.04.2004, p. 1024-1028.

Research output: Contribution to journalArticle

Ruemmele, FM, Jan, DM, Lacaille, F, Cézard, JP, Canioni, D, Phillips, AD, Peuchmaur, M, Aigrain, Y, Brousse, N, Schmitz, J, Revillon, Y & Goulet, O 2004, 'New perspectives for children with microvillous inclusion disease: Early small bowel transplantation', Transplantation, vol. 77, no. 7, pp. 1024-1028. https://doi.org/10.1097/01.TP.0000119163.30745.C1
Ruemmele, Frank M. ; Jan, Dominique M. ; Lacaille, Florence ; Cézard, Jean Pierre ; Canioni, Danielle ; Phillips, Alan D. ; Peuchmaur, Michel ; Aigrain, Yves ; Brousse, Nicole ; Schmitz, Jacques ; Revillon, Yann ; Goulet, Oliver. / New perspectives for children with microvillous inclusion disease : Early small bowel transplantation. In: Transplantation. 2004 ; Vol. 77, No. 7. pp. 1024-1028.
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abstract = "Background. Microvillous inclusion disease (MVID) is a congenital intestinal epithelial cell disorder leading to lifelong intestinal failure. Despite long-term total parenteral nutrition, life expectancy is extremely reduced because of metabolic or septic complications or liver failure. Methods. Twelve patients with early-onset MVID were evaluated between 1995 and 2002 for the possibility of small bowel transplantation (SbTx). Three patients died before they could be placed on the waiting list for SbTx, and one patient is still awaiting SbTx. SbTx was contraindicated in one patient. Results. Seven of 12 patients (six boys and one girl) underwent transplantation (three SbTxs and four combined liver-SbTxs). Actuarial survival rates were 100{\%} and 75{\%} in the SbTx and combined liver-SbTx groups, respectively, with a mean follow-up of 3 years (1.1-8.5 years). In contrast, the survival rate was only 40{\%} in the subgroup of five patients who did not undergo transplantation. After transplantation, all patients were weaned from parenteral nutrition: the five patients with an additional colon graft were weaned within 36 days as opposed to the others without colonic transplant who obtained full intestinal autonomy several months after transplantation. The only two surviving patients who did not undergo SbTx remain highly dependent on total parenteral nutrition, which is complicated by repeated episodes of metabolic decompensation. Conclusions. SbTx alone or in combination with the liver is highly successful in children with MVID, offering them a long-term perspective for the first time. Associated colon grafting markedly improves the outcome and quality of life after SbTx in patients with MVID.",
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T2 - Early small bowel transplantation

AU - Ruemmele, Frank M.

AU - Jan, Dominique M.

AU - Lacaille, Florence

AU - Cézard, Jean Pierre

AU - Canioni, Danielle

AU - Phillips, Alan D.

AU - Peuchmaur, Michel

AU - Aigrain, Yves

AU - Brousse, Nicole

AU - Schmitz, Jacques

AU - Revillon, Yann

AU - Goulet, Oliver

PY - 2004/4/15

Y1 - 2004/4/15

N2 - Background. Microvillous inclusion disease (MVID) is a congenital intestinal epithelial cell disorder leading to lifelong intestinal failure. Despite long-term total parenteral nutrition, life expectancy is extremely reduced because of metabolic or septic complications or liver failure. Methods. Twelve patients with early-onset MVID were evaluated between 1995 and 2002 for the possibility of small bowel transplantation (SbTx). Three patients died before they could be placed on the waiting list for SbTx, and one patient is still awaiting SbTx. SbTx was contraindicated in one patient. Results. Seven of 12 patients (six boys and one girl) underwent transplantation (three SbTxs and four combined liver-SbTxs). Actuarial survival rates were 100% and 75% in the SbTx and combined liver-SbTx groups, respectively, with a mean follow-up of 3 years (1.1-8.5 years). In contrast, the survival rate was only 40% in the subgroup of five patients who did not undergo transplantation. After transplantation, all patients were weaned from parenteral nutrition: the five patients with an additional colon graft were weaned within 36 days as opposed to the others without colonic transplant who obtained full intestinal autonomy several months after transplantation. The only two surviving patients who did not undergo SbTx remain highly dependent on total parenteral nutrition, which is complicated by repeated episodes of metabolic decompensation. Conclusions. SbTx alone or in combination with the liver is highly successful in children with MVID, offering them a long-term perspective for the first time. Associated colon grafting markedly improves the outcome and quality of life after SbTx in patients with MVID.

AB - Background. Microvillous inclusion disease (MVID) is a congenital intestinal epithelial cell disorder leading to lifelong intestinal failure. Despite long-term total parenteral nutrition, life expectancy is extremely reduced because of metabolic or septic complications or liver failure. Methods. Twelve patients with early-onset MVID were evaluated between 1995 and 2002 for the possibility of small bowel transplantation (SbTx). Three patients died before they could be placed on the waiting list for SbTx, and one patient is still awaiting SbTx. SbTx was contraindicated in one patient. Results. Seven of 12 patients (six boys and one girl) underwent transplantation (three SbTxs and four combined liver-SbTxs). Actuarial survival rates were 100% and 75% in the SbTx and combined liver-SbTx groups, respectively, with a mean follow-up of 3 years (1.1-8.5 years). In contrast, the survival rate was only 40% in the subgroup of five patients who did not undergo transplantation. After transplantation, all patients were weaned from parenteral nutrition: the five patients with an additional colon graft were weaned within 36 days as opposed to the others without colonic transplant who obtained full intestinal autonomy several months after transplantation. The only two surviving patients who did not undergo SbTx remain highly dependent on total parenteral nutrition, which is complicated by repeated episodes of metabolic decompensation. Conclusions. SbTx alone or in combination with the liver is highly successful in children with MVID, offering them a long-term perspective for the first time. Associated colon grafting markedly improves the outcome and quality of life after SbTx in patients with MVID.

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