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Hematopoietic Stem Cell Transplantation (HSCT) for Dyskeratosis Congenita(DC): A Review of 12 Patients(pts) Transplanted in Curitiba, Brazil

Track: Poster Abstracts
Saturday, March 1, 2014, 6:45 PM-7:45 PM
Longhorn Hall E (Exhibit Level 1) (Gaylord Texan)
Samantha Nichele, MD , Bone Marrow Transplantation Service, Federal University of Parana, Curitiba, Brazil
Lisandro Ribeiro, MD , Bone Marrow Transplantation Service, Federal University of Parana, Curitiba, Brazil
Gisele Loth, MD , Federal University of Parana, Curitiba, Brazil
Cilmara Kuwahara, MD , Universidade Federal do Paraná, Curitiba, Brazil
Carlos Eduardo Sá Araújo, MD , Federal University of Parana, Curitiba, Brazil
Ana Luiza Melo Rodrigues, MD , Federal University of Parana, Curitiba, Brazil
Marco Bitencourt, MD , Federal University of Parana, Curitiba, Brazil
Adriana Koliski, MD , Federal University of Parana, Curitiba, Brazil
Rodrigo Calado, MD;PhD , Universidade Paulista de Medicina, Ribeirão Preto, Brazil
Mariester Malvezzi, MD. MS , Federal University of Parana, Curitiba, Brazil
Ricardo Pasquini, MD , Internal Medicine, Federal University of Parana, Curitiba, ., Brazil
Rebeca Toassa Mousquer, MD , Federal University of Parana, Curitiba, Brazil
Carmem Bonfim, MD , Federal University of Parana, Curitiba, Brazil
DC is an inherited bone marrow failure syndrome (BMF) with a high predisposition to cancer development. The HSCT is the only curative treatment for BMF associated but imposes significant toxicity. We reported the outcome of 12 pts who underwent bone marrow transplantation for DC between July 1993 and May 2013 in a single institution. The median age at transplantation was 16,5 years (range 3 to 27). Two pts had the non classical forms of disease (Revesz syndrome and severe aplastic anemia with evidence of short telomeres). Five pts received bone marrow from their HLA matched siblings and were conditioned with Cyclophosphamide (CY) 200mg/Kg. Seven pts were transplanted  from unrelated donors (URD): 6 BM (match 10/10: 4pts and 9/10: 2pts) and 1 CB (5/6). Conditioning Regimen for the URD BMT was CY30-60mg/Kg + Fludarabine125mg/m2 + rabbitATG5mg/Kg and CB Busulfan12mg/Kg + Fludarabine125mg/m2 + rATG5mg/Kg. Graft versus host disease (GVHD) prophylaxis: methotrexate and cyclosporine for all patients, except for the one who received CBU. Median of TNC: BM: 3.97 x 108/Kg. CBU: 6.5 x107/Kg. All pts survived more than 28 days and were available for engraftment. All but one pt had stable hematological engraftment. Chimerism was complete in 7 patients and mixed in the other 5 patients. Mucositis was the only toxicity observed (grade II in 5 pts and grade III in 2 pts). GVHD was observed only among unrelated transplants. Two pts developed grade II acute GVHD and both progressed to chronic GVHD (limited). The probability of survival in 2 year was 50%. Two deaths were related to the procedure (SOS and adenovirus sepsis). Four pts died between 1 and 10 years after transplant because of progression of underlying disease. Six patients (4 recipients of an URD) are alive between 6 months and 11 years after transplant with a median follow up of 1.6 years. Conclusions: Regimen related toxicity was very low and an appropriate engraftment was achieved. Late mortality was consequent to the progression of underlying disease. Long term follow up is essential in order to detect late complications related to the transplant procedure or the underlying disease.
Disclosures:
Nothing To Disclose
See more of: Poster Session 2: Allogeneic Transplants
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