الفهرس 
APLASTIC ANEMIAOnly 14 pages are availabe for public view
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Abstract
A
plastic anemia (AA) is a syndrome of peripheral blood pancytopenia and bone marrow hypoplasia in the absence of major dysplasia, a malignant infiltrate or fibrosis. Clinical course of AA was found to be dependent on the degree of aplasia. In severe forms of the disease, rapid deterioration of patient’s condition and possible incidence of fatal complications is the rule.
HSCT from a matched sibling donor is the treatment of choice for children and young adults with severe aplastic anemia. However, it has several acute and chronic complications.
Late complications and effects after transplantation became an increasingly important aspect of management. The aim of this work is to study the outcome and late effects among children with severe AA who received MRD peripheral blood stem cells PBSC transplantation and Cytoxan and Antithymocyte globulin conditioning regimen at Ain Shams University Pediatric BMT Unit.
After obtaining informed consent, we analyzed data of 15 patients with severe aplastic anemia received matched related peripheral blood stem cells transplantation between January 2011 and December 2018.
All long- term survivors were subjected to the following assessment: Thorough history and clinical examination with detailed assessment of growth and development, symptoms, and signs of chronic GvHD in their follow-up visits
Investigations for the graft included CBC with differential, reticulocyte counts and chimerism.
Evaluation of immune reconstitution (IR) was done by flow-cytometry for CD markers (4, 8, 19 & 56) and by quantification of serum total IgG, IgM, and IgA.
Hepatic function was assessed by alkaline phosphatase, liver enzymes, albumin, Lactate dehydrogenase and total bilirubin, as well as serum ferritin for iron overload.
Renal function tests included serum creatinine and blood urea nitrogen.
Lipid Profile and random blood sugar were measured.
Thyroid Functions by thyroid stimulating hormone (TSH), Free thyroxine (FT4), Free triiodothyronine (FT3) and gonadal function in adolescent patients by Follicle-stimulating hormone (FSH) and Luteinizing hormone (LH). Spirometry and echocardiography were used to follow up pulmonary and cardiac function respectively.
Patients were also evaluated for infectious complications and secondary lymphoma or solid tumor.
The median duration of the study was 87 months (43-115 months). The overall survival was 73.3%. Event free survival was 66.7%. All mortality was within 2 months post-transplant.
Blood counts were within normal limits. Chimerism was complete for all except one. Immune reconstitution was consistent.
Growth assessment revealed Z score of weight, height, and body mass index 0(-1.8-1.6), -0.7 (-1.8-0.95) and 0.34 (-1.29-2). Abnormal thyroid function was found in 3 patients (27%).
No documented high grade acute graft-versus-host disease or extensive chronic graft-versus-host disease.
Cardiac, pulmonary, hepatic and renal functions showed no abnormality, and no malignant complications were reported.
Our study is limited with the small number of patients underwent HSCT for aplastic anemia in our unit, but the long-term follow-up added to its strength. And further studies are still needed on bigger number of cases.
CONCLUSION
I
n conclusion, MRD PBSCT and Cytoxan/ATG conditioning is the preferred way of transplant in aplastic anemia in a developing country like Egypt. It is associated with good OS and EFS. Moreover, long term survivors experience very good quality of life with normal growth and normal hematopoiesis and fast good immune reconstitution. However, follow up studies is essential for early detection and immediate intervention to minimize the burden of morbidity on long-term after HSCT.
RECOMMENDATIONS
Early diagnosis of AA patients and early referral to highly specialised centre.
Early HLA matching for proper and early intervention for early transplant.
HSCT from an HLA-matched donor offers definitive curative treatment.
Thorough assessment of patients and donors before transplantation
PBSC-Transplantation and CY/ATG conditioning is the preferred way of transplantation in AA in a developing country like Egypt. It is associated with good OS and EFS.
It’s recommended to continue following up the patients of AA even after successful transplantation for early recognition and management of late effects of transplantation.
Further studies on larger populations of LTS to quantify the burden of morbidity borne by HSCT survivors and identify those at risk, emphasizing the need for close monitoring of these vulnerable populations.