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| Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002 |
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| Author(s): Gennery AR, Khawaja K, Veys P, Bredius RGM, Notarangelo LD, Mazzolari E, Fischer A, Landais P, Cavazzana-Calvo M, Friedrich W, Fasth A, Wulffraat NM, Matthes-Martin S, Bensoussan D, Bordigoni P, Lange A, Pagliuca A, Andolina M, Cant AJ, Davies EG |
| Source: BLOOD Volume: 103 Issue: 3 Pages: 1152-1157 Published: FEB 1 2004 |
| Times Cited: 23 References: 27 |
| Abstract: CD40 ligand (CD40L) deficiency causes recurrent sinopulmonary infection, Pneumocystis carinii pneumonia, and Cryptosporidium parvum infection. Approximately 40% to 50% of patients survive to the third decade: long-term survival is unclear. Hematopoietic stem cell transplantation (HSCT) is curative. We present a retrospective analysis of 38 European patients undergoing HSCT for CD40L deficiency in 8 European countries between 1993 and 2002. Donor stem cell source included 14 HLA-identical siblings, 22 unrelated donors, and 2 phenotypically matched parental stem cells (12 T-cell depleted). Of the patients, 34 engrafted and 26 (68%) survived; 3 had autologous reconstitution, 22 (58%) were cured, and 1 engrafted but has poor T-cell immune reconstitution. There were 18 evaluated patients who responded to vaccination. Of the patients, 12 (32%) died from infection-related complications, with severe cryptosporidiosis in 6. Grades 2 to 4 graft-versus-host disease (GvHD) associated with infection occurred in 6 of 12 fatal cases. HSCT cured 58% of patients, 72% of those without hepatic disease. Early T-cell function following whole marrow HSCT may limit cryptosporidial disease, but survival was similar after T-cell-depleted HSCT. Preexisting lung damage was the most important adverse risk factor. Further studies will determine optimal timing and type of HSCT. |
| Document Type: Article |
| Language: English |
| Reprint Address: Gennery, AR (reprint author), Newcastle Gen Hosp, Westgate Rd, Newcastle Upon Tyne NE4 6BE, Tyne & Wear England |
Addresses:
1. Newcastle Gen Hosp, Newcastle Upon Tyne NE4 6BE, Tyne & Wear England
2. Great Ormond St Hosp Sick Children, London WC1N 3JH, England
3. Leiden Univ, Ctr Med, Leiden, Netherlands
4. Univ Brescia, I-25121 Brescia, Italy
5. Hop Necker Enfants Malad, Paris, France
6. Univ Ulm, Childrens Hosp, Ulm, Germany
7. Univ Gothenburg, Gothenburg, Sweden
8. Wilhelmina Childrens Hosp, Utrecht, Netherlands
9. St Anna Childrens Hosp, A-1090 Vienna, Austria
10. Childrens Hosp, Vandoeuvre Les Nancy, France
11. Lower Silesian Ctr Cellular Transplantat, Wroclaw, Poland
12. Kings Coll Hosp, London, England
13. IRCCS, Pediat Clin, Trieste, Italy |
| Publisher: AMER SOC HEMATOLOGY, 1900 M STREET. NW SUITE 200, WASHINGTON, DC 20036 USA |
| Subject Category: Hematology |
| IDS Number: 766UK |
| ISSN: 0006-4971 |
| DOI: 10.1182/blood-2003-06-2014 |
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