scholarly journals Haploidentical bone marrow transplantation improves cerebral hemodynamics in adults with sickle cell disease

2019 ◽  
Vol 94 (6) ◽  
pp. E155-E158 ◽  
Author(s):  
Lori C. Jordan ◽  
Meher R. Juttukonda ◽  
Adetola A. Kassim ◽  
Michael R. DeBaun ◽  
Larry T. Davis ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Marrow Transplantation ◽  
Cerebral Hemodynamics ◽  
Cell Disease

scholarly journals Pulmonary, Gonadal, and Central Nervous System Status after Bone Marrow Transplantation for Sickle Cell Disease

2010 ◽  
Vol 16 (2) ◽  
pp. 263-272 ◽  
Author(s):  
Mark C. Walters ◽  
Karen Hardy ◽  
Sandie Edwards ◽  
Thomas Adamkiewicz ◽  
James Barkovich ◽  
...  
Keyword(s):  
Central Nervous System ◽  
Bone Marrow ◽  
Nervous System ◽  
Bone Marrow Transplantation ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Marrow Transplantation ◽  
Cell Disease

Iron Overload in Acute Myelogenous Leukemia after Bone Marrow Transplantation.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 5336-5336
Author(s):  
Zahra Pakbaz ◽  
Roland Fischer ◽  
Nancy Noonan ◽  
Sherrie Shiota ◽  
Paul Harmatz ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Sickle Cell Disease ◽  
Iron Overload ◽  
Sickle Cell ◽  
Serum Ferritin ◽  
Marrow Transplantation ◽  
Cell Disease ◽  
Acute Leukemias ◽  
Wet Weight

Abstract Children with acute leukemias typically receive RBC transfusions during the course of their treatment. However, the severity and significance of transfusional iron overload is not known in this patient population. Earlier, we reported elevated serum ferritin (SF) in 5 patients with AML who received HLA-identical sibling bone marrow transplantation (BMT). However, SF has a wide predictive interval for liver iron concentration (LIC) in thalassemia and sickle cell disease and the current recommendation is to measure LIC to estimate total body iron burden. Further exploration of the SF-to-LIC ratio (SF/LIC) to investigate the relationship between SF and LIC has shown ratio differences by specific disease (SCD, thalassemia, genetic hemochromatosis), transfusion status and use of chelation. The reasons for these differences are not presently known. In this study LIC was measured within 2 weeks of serum ferritin (SF), in 8 AML patients after transplantation, to explore the significance of the elevated SF and to determine the range and character of the SF/LIC ratio after BMT for AML. LIC was measured (1–4 year after BMT) by a low temperature SQUID biosusceptometer system (Ferritometer®) under the standardized Hamburg-Torino-Oakland protocol. The range for LIC in healthy individuals measured by SQUID is 90–340 mg/g wet weight. The median serum ferritin was 1227 (582–1723) μg/l and the median LIC was 1284 (751–1612) mg/g wet weight or approximately 4 times greater than the upper limit of normal. ALT was measured in 4 patients of which 2 were mildly elevated. Neither LIC nor SF changed over the interval of follow-up extending to 3 years in 2 patients (aged 11.5y and 14.5 y) who returned annually for LIC measurements. The ratio of SF/LIC ranged from 0.5 to 1.4 (median: 0.9) in the patients with AML. This compares to ratios of 1.2 (0.6–2.6) in regularly transfused sickle cell disease patients (n=45), 0.87 (0.23–2.7) in transfusion dependent thalassemia patients and 0.32 (0.05–0.57) in transfusion independent thalassemia patients. These preliminary observations suggest that children with acute leukemias who undergo bone marrow transplantation develop significant transfusion related iron accumulation. Additional investigation should be undertaken to determine if AML patients would benefit from iron reduction therapy by phlebotomy after BMT.

Bone Marrow Transplantation without Myeloablation for Sickle Cell Disease

2001 ◽  
Vol 344 (1) ◽  
pp. 68-68 ◽  
Author(s):  
Lakshmanan Krishnamurti ◽  
Bruce R. Blazar ◽  
John E. Wagner
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Marrow Transplantation ◽  
Cell Disease

Bone-Marrow Transplantation in Sickle-Cell Disease

1997 ◽  
Vol 79 (11) ◽  
pp. 1726-1730 ◽  
Author(s):  
P. Hernigou ◽  
F. Bernaudin ◽  
P. Reinert ◽  
M. Kuentz ◽  
J. P. Vernant
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Marrow Transplantation ◽  
Cell Disease

Fludarabine/Treosulfan/Thiotepa/ATG Conditioning for Matched and Single Antigen Mismatched Unrelated Bone Marrow Transplantation in Haemoglobinopathies Is Feasible and Leads to Early and Sustanined Engraftment with No Long-Term Toxicity or GvHD

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2468-2468
Author(s):  
Farah O'Boyle ◽  
Leena Karnik ◽  
Anne Bradshaw ◽  
Richard M Szydlo ◽  
Josu de la Fuente
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Marrow Transplantation ◽  
Graft Failure ◽  
Acute Gvhd ◽  
Thalassaemia Major ◽  
Cell Disease

Abstract Related haemopoietic stem cell transplantation is a well-established treatment modality for haemoglobinopathies, but it is limited by the availability of related donors. The results of unrelated transplantation have been variable with historical variable results, concern about GvHD and usually restricted to 10/10 matches. From 2011 to 2016 twelve consecutive unrelated bonemarrow transplants were conditioned with fludarabine 160 mg/m2, treosulfan 42 g/m2, thiotepa 10 mg/kg and ATG (Thymoglobulin) 11.25 mg/kg (FTTA). Endogenous haemopoiesis was suppressed pre-transplantation with hypertransfusions for a minimum of 8 weeks. GvHD prophylaxis was provided with ciclosporin and MMF. 7 patients were transplanted for β thalassaemia major, one of α thalassaemia major and 4 for sickle cell disease. The median age was 9.5 years (2 - 17). The source of stem cells was BM in all patients: five 10/10 matched, six 9/10 matched and one 11/12 matched. The median cell dose was 3.01 x 108 TNC/kg (range 1.53 - 11.07) and 4.27 x 106 CD34+/kg (range 1.16 - 27.41). The median survival was 9.9 months (6.9-44.1). Patients with thalassaemia were Pesaro class I or II (Pesaro class III patients were intensively chelated pre-transplantation to return to class I or II). Patients with sickle cell disease were transplanted for stroke or recurrent vaso-oclusive crises and/or acute chest syndrome not responding to hydroxycarbamide. All patients were evaluated with liver biopsy pre-transplantation and defibrotide prophylaxis given if Ishak stage ≥3. All patients engrafted and achieved evidence of donor haemopoiesis on day +28 and achieved transfusion-independence and donor haematological values. No patient suffered primary or secondary graft failure. There was one death, one on day +257 due to idiopathic pneumonia syndrome in a patient with b thalassaemia major. Acute GvHD ≥ grade 2 occurred in 5 patients (41.7%). Chronic limited GvHD did not occur in any patient, but extensive occurred in the 5 patients developing acute GvHD. None of the patients had chronic GvHD at 18 months. VOD occurred in 4 patients (33.3%) and responded to standard measures and defibrotide treatment. The median neutrophil engraftment was 13 days (range 9 to 22). Patient with sickle cell disease had the platelet count maintained >50 x 109/L at all times. The median platelet engraftment >50 x 109/L was 37 days (range 15 to 86) and >50 x 109/L was 32 days (range 15 to 111). The median time to cessation of immunosuppression was 134 days (68-219). Chimerism studies on day +28 demonstrated 100% in whole blood (WB) and 88.9% in T cells (T) >95%, and 0% WB and 11.1% T >50-89% [n=11]; day +90: 91.7% WB and 91.7% T >95%, 8.3% WB and 0% T >50-89%, 0% WB and 8.3% T <50% [n=12]; day +180: 91.7% WB and 91.7% T >95%, and 8.3% WB and 8.3% T >50-89% [n=12]; and day +365: 50% WB and 50% T >95%, 25% WB and 25% T >90-95%, and 25% WB and 25% T >50-89% [n=4]. In conclusion, unrelated bone marrow transplantation for all haemoglobinopathies is feasible and the overall survival and disease-free survival is 90.9%. FTTA leads to early and sustained engraftment with low rate of graft failure, and whilst the occurrence of VOD despite adequate chelation pre-transplantation and the incidence of GvHD was significant, these complications resolved with standard therapies and long-term outcomes approach those of related transplantation with no treatment required by 18 months. Disclosures No relevant conflicts of interest to declare.

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