PMID- 16339666
OWN - NLM
STAT- MEDLINE
DCOM- 20060713
LR  - 20191210
IS  - 0077-8923 (Print)
IS  - 0077-8923 (Linking)
VI  - 1054
DP  - 2005
TI  - Bone marrow transplantation in adults with thalassemia: Treatment and long-term 
      follow-up.
PG  - 196-205
AB  - Current regular blood transfusion programs and chelation treatment have 
      considerably improved survival of patients with thalassemia, which resulted in a 
      larger proportion of adult patients. However, disease- and treatment-related 
      complications in these patients progress over time, causing severe morbidity and 
      shortened life expectancy. Stem cell transplantation still remains the only cure 
      currently available for patients with thalassemia. This study updates transplant 
      outcomes in 107 adult patients with median age of 22 years (range, 17-35 years) 
      who received bone marrow transplantation (BMT) from human leukocyte antigen 
      (HLA)-identical related donors between 1988 and 1996 (group A) and describes the 
      results of BMT in 15 adult patients with median age of 21 years (range, 17-31 
      years) who were treated with a new treatment protocol (Protocol 26) between 1997 
      and 2003 (group B). The probability of survival, event-free survival, 
      nonrejection mortality, and rejection for group A patients were 66%, 62%, 37%, 
      and 4%, respectively, with a median follow-up of 12 years (range, 8.3-16.2 
      years). Group B patients treated with the new protocol had some improvement in 
      thalassemia-free survival (67%) and lower transplant-related mortality (27%) than 
      that of previous protocols. However, transplant-related mortality in these 
      high-risk patients remains elevated. Current myeloablative BMT in adult patients 
      is characterized by higher transplant-related toxicity due to an advanced phase 
      of disease. Although this new approach to transplant adult patients with a 
      reduced-dose intensity-conditioning regimen has improved thalassemia-free 
      survival, transplant-related mortality in these high-risk patients remains 
      elevated.
FAU - Gaziev, Javid
AU  - Gaziev J
AD  - Mediterranean Institute of Hematology, International Center for Transplantation 
      in Thalassemia and Sickle Cell Anemia, Policlinico Tor Vergata, Rome, Italy. 
      j.gaziev@fondazioneime.org
FAU - Sodani, Pietro
AU  - Sodani P
FAU - Polchi, Paola
AU  - Polchi P
FAU - Andreani, Marco
AU  - Andreani M
FAU - Lucarelli, Guido
AU  - Lucarelli G
LA  - eng
PT  - Evaluation Study
PT  - Journal Article
PL  - United States
TA  - Ann N Y Acad Sci
JT  - Annals of the New York Academy of Sciences
JID - 7506858
RN  - 0 (Hematopoietic Cell Growth Factors)
RN  - 0 (Immunosuppressive Agents)
RN  - 0 (Iron Chelating Agents)
RN  - 11096-26-7 (Erythropoietin)
RN  - 143011-72-7 (Granulocyte Colony-Stimulating Factor)
RN  - FA2DM6879K (Vidarabine)
RN  - G1LN9045DK (Busulfan)
RN  - J06Y7MXW4D (Deferoxamine)
RN  - MRK240IY2L (Azathioprine)
RN  - P2K93U8740 (fludarabine)
RN  - X6Q56QN5QC (Hydroxyurea)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Azathioprine/administration & dosage
MH  - Bone Marrow Transplantation/adverse effects/mortality/*statistics & numerical 
      data
MH  - Busulfan/administration & dosage
MH  - Chelation Therapy
MH  - Clinical Protocols
MH  - Combined Modality Therapy
MH  - Comorbidity
MH  - Deferoxamine/therapeutic use
MH  - Disease-Free Survival
MH  - Erythrocyte Transfusion
MH  - Erythropoietin/administration & dosage
MH  - Female
MH  - Follow-Up Studies
MH  - Graft vs Host Disease/epidemiology/etiology/prevention & control
MH  - Granulocyte Colony-Stimulating Factor/administration & dosage
MH  - Hematopoietic Cell Growth Factors/administration & dosage
MH  - Hemosiderosis/epidemiology/etiology/therapy
MH  - Humans
MH  - Hydroxyurea/administration & dosage
MH  - Immunosuppressive Agents/administration & dosage
MH  - Iron Chelating Agents/therapeutic use
MH  - Life Tables
MH  - Liver Cirrhosis/complications
MH  - Male
MH  - Phlebotomy
MH  - Postoperative Complications/mortality
MH  - Survival Analysis
MH  - Thalassemia/complications/drug therapy/mortality/*surgery/therapy
MH  - Transfusion Reaction
MH  - Transplantation Conditioning/methods/mortality/statistics & numerical data
MH  - Transplantation, Homologous/adverse effects/mortality/statistics & numerical data
MH  - Treatment Outcome
MH  - Vidarabine/administration & dosage/analogs & derivatives
EDAT- 2005/12/13 09:00
MHDA- 2006/07/14 09:00
CRDT- 2005/12/13 09:00
PHST- 2005/12/13 09:00 [pubmed]
PHST- 2006/07/14 09:00 [medline]
PHST- 2005/12/13 09:00 [entrez]
AID - 1054/1/196 [pii]
AID - 10.1196/annals.1345.024 [doi]
PST - ppublish
SO  - Ann N Y Acad Sci. 2005;1054:196-205. doi: 10.1196/annals.1345.024.