Case Reports LATE VASCULAR COMPLICATIONS AFTER BONE MARROW TRANSPLANTATION FOR DYSKERATOSIS CONGENITA

Berthou, Christian; Devergié, A; D'Agay, Marie Francloise; Sonsino, E; Scrobohaci, Marie-Lorraine; Loirat, Chantal; Gluckman, Éliane

doi:10.1111/j.1365-2141.1991.tb04543.x

Cited by 40 publications

(44 citation statements)

References 5 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Previous reports (Table 3) have described progression of the clinical signs of DKC after transplantation, which was indistinguishable from the natural course of disease. [8][9][10][11][20][21][22][23] In our series, all patients except one received low intensity conditioning regimens. The conditioning protocol was different across different centers.…”

Section: Discussionmentioning

confidence: 85%

“…Reduced intensity preparative regimens are less likely to cause organ toxicity and may improve long-term outcomes. [9][10][11][12] CASE SUMMARIES AND RESULTS Herein, we review nine patients who received allo-SCT at five centers from five different member countries in the Eastern Mediterranean Blood and Marrow Transplantation Registry. Two of these cases were previously published as separate case reports and one as a brief letter.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenita-related BM failure

Ayas

Nassar

Hamidieh

et al. 2013

Bone Marrow Transplant

View full text Add to dashboard Cite

Section: Discussionmentioning

confidence: 85%

Section: Introductionmentioning

confidence: 99%

Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenita-related BM failure

Ayas

Nassar

Hamidieh

et al. 2013

Bone Marrow Transplant

View full text Add to dashboard Cite

“…In previous reports in Fanconi's anemia, cyclophosphamide with or without thoraco-abdominal irradiation was used 12,13 and there is little experience with busulfan. In addition, irradiation may cause late vascular complications after bone marrow transplantation for dyskeratosis 7 and we suggest that the good results obtained by our team is due to avoidance of TBI in conditioning. [6][7][8][9][10] In our patients reduced dose of busulfan was well tolerated with no major side-effects.…”

Section: Discussionmentioning

confidence: 78%

“…In addition, irradiation may cause late vascular complications after bone marrow transplantation for dyskeratosis 7 and we suggest that the good results obtained by our team is due to avoidance of TBI in conditioning. [6][7][8][9][10] In our patients reduced dose of busulfan was well tolerated with no major side-effects. Also, differentiation of skin and GI lesions of this disease from chronic GVHD is not possible.…”

Section: Discussionmentioning

confidence: 78%

“…Results of allogeneic bone marrow transplantation in these patients have been relatively poor due to early and late complications. [6][7][8][9][10] We describe the effect of bone marrow transplantation for aplastic anemia in two siblings with dyskeratosis congenita from a single donor. The other brother of these two siblings had died due to bone marrow failure and dyskeratosis congenita previously.…”

mentioning

confidence: 99%

See 1 more Smart Citation

Correction of bone marrow failure in dyskeratosis congenita by bone marrow transplantation

Ghavamzadeh

Alimoghadam

Nasseri

et al. 1999

Bone Marrow Transplant

View full text Add to dashboard Cite

Summary:Dyskeratosis congenita is recognized by its dermal lesions and constitutional aplastic anemia in some cases. We report successful allogeneic bone marrow transplantation in two siblings with this disease from their sister, and their long term follow-up. We used reduced doses of cyclophosphamide and busulfan for conditioning instead of total body irradiation. Also, we report late adverse effects of transplantation which are not distinguishable from the natural course of disease. Keywords: allogeneic bone marrow transplantation; constitutional anemia; dyskeratosis congenita Dyskeratosis congenita is a rare congenital syndrome characterized by atrophy and reticular pigmentation of skin, dystrophy of nails, 1 leukoplakia together with multi-system ectodermal and some mesodermal changes. Skin changes are typically located on the upper torso, neck and face, although the extremities may also be involved. Other manifestations of dyskeratosis congenita may be some other epithelial changes such as gingival disorders, dysphagia due to esophageal strictures, skeletal abnormality, aplastic anemia, 2 mental deficiency and hypersplenism.Severe aplastic anemia appears between the ages of 5 and 10 years. 3 Most of the cases transmit as an X-linked recessive inheritance pattern, although autosomal recessive 4 and autosomal dominant transmission 5 have also been reported. Results of allogeneic bone marrow transplantation in these patients have been relatively poor due to early and late complications. [6][7][8][9][10] We describe the effect of bone marrow transplantation for aplastic anemia in two siblings with dyskeratosis congenita from a single donor. The other brother of these two siblings had died due to bone marrow failure and dyskeratosis congenita previously. The parents of patients were not first cousins and were phenotypically normal.Correspondence: Dr K Alimoghadam, Shariati Hospital, Karger Aven, Tehran 14114 Iran Received 3 August 1998; accepted 8 September 1998Case report Case 1Patient 1 was an 18-year-old man with dyskeratosis congenita with reticular pigmentation, nail dystrophy, leukoplakia and marrow failure. He had received oxymetholone before BMT, with a history of frequent blood and platelet transfusion. Pulmonary function tests before bone marrow transplantation were normal. His bone marrow was hypoplastic and peripheral blood indices showed pancytopenia (Table 1).His conditioning regimen was cyclophosphamide 20 mg/kg/day for 4 days and busulfan 0.2 mg/kg/day for 4 days. Transplantation was carried out with infusion of 4.6 × 10 8 mononuclear cells per kilogram of body weight from bone marrow of his HLA-identical healthy sister. We used methotrexate and cyclosporin for GVHD prophylaxis and mild skin and gastrointestinal acute GVHD occurred after transplantation. He was discharged from hospital 37 days after marrow transplantation. On further follow-up, only his skin showed erythematous lesions and desquamation of his face, which was not possible to differentiate between chronic GVHD and dyskeratosis. He ...

show abstract

Inherited Bone Marrow Failure Syndromes

Dror¹

2006

Pediatric Hematology

View full text Add to dashboard Cite

Case Reports LATE VASCULAR COMPLICATIONS AFTER BONE MARROW TRANSPLANTATION FOR DYSKERATOSIS CONGENITA

Cited by 40 publications

References 5 publications

Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenita-related BM failure

Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenita-related BM failure

Correction of bone marrow failure in dyskeratosis congenita by bone marrow transplantation

Inherited Bone Marrow Failure Syndromes

Contact Info

Product

Resources

About