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ABSTRACT: Glanzmann thrombasthenia (GT) is a rare autosomal recessive bleeding disorder characterized by normal platelet count, but lack of platelet aggregation. The molecular basis is linked to quantitative and/or qualitative abnormalities of the membrane glycoprotein IIb/IIIa complexes. Usually it is associated with mild bleeding but may lead to severe and potentially fatal hemorrhages. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment. However, because of the risks associated with HSCT, it is generally not recommended unless there are life threatening hemorrhages, or the patient has developed refractoriness to platelet transfusion due to antibody formation. Herein, we report an 11-year-old female from United Arab Emirates (UAE) with severe GT and anti platelet alloimmunization successfully treated with HSCT from her HLA-identical sibling.
Pediatric Blood & Cancer 01/2009; 52(5):682-3. · 1.89 Impact Factor
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ABSTRACT: We reviewed 26 consecutive patients with AML who were transplanted in second CR2 between 1994 and 2005. The most common conditioning regimen was CY and TBI. Median age at transplant was 8.9 yr (range 2.2-18.2). Nine patients received related donor, 16 patients received unrelated donors, and one patient received unrelated cord stem cells. Acute grade III-IV and chronic extensive GVHD occurred in eight (30%) and nine (35%) patients, respectively. Six patients (23%) relapsed, four of them died. Six patients (23%) died from TRM. Estimate of three-yr EFS was 0.53 (95% CI; 0.34-0.72). Including the two relapsed patients who were salvaged by DLI and second transplantation, three-yr OS was 0.61 (95% CI; 0.41-0.78) with a median follow-up of three and a half yr (range 1.5-11.2 yr). When entering remission, children with relapsed AML have a reasonable survival with HSCT, but relapse and TRM remain a concern.
Pediatric Transplantation 12/2008; 13(8):999-1003. · 1.48 Impact Factor
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ABSTRACT: A 15-year-old male with beta-thalassemia major developed dilated cardiomyopathy secondary to iron-overload (Z-scores of left ventricle (LV) dimensions >3, ejection fraction (EF) 33%). Treatment with deferoxamine was unsuccessful, presumably due to poor compliance. After 15 months of using deferasirox (DFX), LV end-diastolic dimension normalized (Z-scores <2), and EF improved to 58%. We conclude that treatment with DFX resulted in a reversal of iron-induced cardiomyopathy.
Pediatric Blood & Cancer 12/2008; 52(3):426-8. · 1.89 Impact Factor
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ABSTRACT: We reviewed 70 consecutive children with AML who received hematopoietic stem cell transplantation (HSCT) in our institution between 1994 and 2005. Forty-seven children were transplanted in CR1 and 23 were transplanted in CR2. BU/CY was the most common pretransplant conditioning regimen for CR1 patients and a TBI-based conditioning regimen was the most common regimen for CR2 patients. Most patients transplanted in CR1 (81%) received related donor HSCT, whereas most of the CR2 patients (74%) received unrelated donor HSCT. Expectedly, there was a significant increase in acute GVHD incidence in CR2 patients (40 vs 25% for grades I-II and 30 vs 10% for grades III-IV; P=0.02) and a significant increase in transplant-related mortality (38 vs 11%; P=0.01). Although the difference between 3-year EFS for CR1 and CR2 was not statistically significant, there was a significantly superior 3-year overall survival for CR1 patients (74 vs 51%; P=0.05). Children with relapsed AML who achieve and maintain remission until HSCT, have a reasonable survival, but the outcome of children receiving HSCT in CR1 remains superior.
Bone Marrow Transplantation 07/2008; 41(11):941-5. · 3.75 Impact Factor
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ABSTRACT: Children with acute lymphoblastic leukaemia (ALL) receiving haematopoietic stem cell transplantation (HSCT) in third complete remission (CR3) are at high risk for transplant-related mortality (TRM) and relapse. Twenty-two consecutive children with ALL in CR3 received HSCT between January 1994 and August 2005. Ten patients died of TRM, seven patients relapsed, six did not have graft-versus-host disease (GVHD). Five patients were long-term survivors, (median follow-up, 5.8 years; range 2.9-11.7). Three-year event-free survival was 0.32 (95% confidence interval 0.19 and 0.59). Survivors had moderate to severe GVHD. Allowing some GVHD or exploring means of inducing GVHD should be considered in CR3 patients.
British Journal of Haematology 02/2008; 140(1):86-9. · 4.94 Impact Factor
