HA-03. Allo-HSCT in sickle cell anemia and thalassemia: a case presentation
Lusine M. Krmoyan1, Mane S. Gizhlaryan1, Karen H. Meliksetyan1, Narine A. Ghazaryan1, Dianna R. Soghomonyan1, Nelli O. Musayelyan1, Armine A. Pepanyan1, Andranik G. Shamilyan1, Vahe A. Mayilyan1, Inga V. Khalatyan1, Taguhi J. Hovhannisyan1, Melanya S. Sahakyan1, Emma Tadevosyan1, Gevorg N. Tamamyan1, Samvel H. Danielyan1, Lawrence Faulkner2
1 R. H.Yeolyan Hematology Center, Yerevan, Armenia
2 Cure2Children Foundation, Florence, Italy
Contact: Dr. Lusine M. Krmoyan, phone: (+374) 948 09427, (+374) 913 87474, e-mail: email@example.com
Hematopoietic stem cell transplantation (HSCT) is an effective treatment option for many children hereditary diseases of the immune or hematopoietic system, as well as ones with malignant neoplasms. The organization and conduct of HSCT are difficult and costly and not all countries have the resources and trained personnel to establish a HSCT program. Nevertheless, in 2016, a HSCT department with 7 beds was opened at the Hematology Center named after Prof. R. Yeolyan (HC), where auto-HSCT was performed up until 2021. A total of 75 auto-HSCTs were performed, 19 of which were in children. In April 2021, an allogeneic HSCT project was launched at the HC in the framework of cooperation with the Ministry of Health, the City of Smile Foundation (Armenia), DKMS (Germany) and Cure2Children (Italy). The main goal of the project is to develop allogeneic HSCT in Armenia and offer free or highly subsidized HSCT to African children with Sickle cell anemia (SCA) under the supervision of international HSCT experts.
Materials and methods
SCA with frequent crises and severe thalassemia is curable with HSCT resulting in normalization of hematopoiesis and disease symptoms attenuation resulting in quality of life improvement. The first two patients who underwent HSCT at HC were SCA patients with HLA-matched related donors. Prior to starting the conditioning regimen and placing a central catheter, exchange transfusions were performed in order to reduce HbS to <30%. Also, 10 ml/kg of the patient’s blood was removed within 1 hour, followed by an exchange transfusion at the rate of 15 ml/kg within the next three hours. Myeloablative conditioning regimen including Fludarabine 30 mg/m2/day from day -18 to -13 (total 180 mg/m2), ATG (Rabbit) at total dose of 4 mg/kg on day -12 to -10, Busulfan 14 mg/kg given on day -9 to -6, Cyclophosphamide 200 mg/kg/day from day -5 to -2 followed by the infusion of freshly harvested HLA-compatible G-CSF-primed marrow on day 0. Graft-versus-host disease (GVHD) prophylaxis consists of Cyclosporine A and Methotrexate. Chimerism is evaluated on D+30, D+60, and D+ 90. In order to evaluate the results of transplantation, hemoglobin electrophoresis is also performed.
The patient is a 9 years old girl from Nigeria with SCA who was admitted to the HC in September 2021 with complaints of frequent abdominal pain, mucosal icterus. The patient’s donor was a match related sibling. The child received hydroxyurea prior to admission to the HC at a dose of 20-30 mg/kg/day, considering hepatosplenomegaly. During the examination in the HC, before HSCT, was diagnosed with bilirubinemia (total bilirubin 60.0-80.0 µmol/l, indirect bilirubin 54.0-70.0 µmol/l). A blood test was conducted at the Genetic Center and a diagnosis of Gilbert’s syndrome was made. After consultation with experts it was decided to perform HSCT according to the protocol without reducing drug doses or changing conditioning regimen. The transplantation proceeded without complications. Engraftment was on D +20. Bilirubin began to decrease immediately after D0. The patient developed grade 1 aGVHD of the skin and GIT, which resolved without further treatment. Currently, the child’s condition is stable; the dose of Cyclosporine is tapered.
This case illustrates our experience of allo-HSCT performed in a patient with SSA.
Sickle-cell anemia, thalassemia, children, allo-HSCT, Armenia.