scholarly journals Successful hematopoietic stem cell transplantation in a patient with congenital dyserythropoietic anemia type II

2014 ◽  
Vol 18 (4) ◽  
pp. E130-E133 ◽  
Author(s):  
Sule Unal ◽  
Roberta Russo ◽  
Fatma Gumruk ◽  
Baris Kuskonmaz ◽  
Mualla Cetin ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Type Ii ◽  
Hematopoietic Stem ◽  
Congenital Dyserythropoietic Anemia

scholarly journals Successful Allogeneic Hematopoietic Stem Cell Transplantation of a Patient Suffering from Type II Congenital Dyserythropoietic Anemia A Rare Case Report from Western India

2015 ◽  
Vol 2015 ◽  
pp. 1-4 ◽  
Author(s):  
Gaurang Modi ◽  
Sandip Shah ◽  
Irappa Madabhavi ◽  
Harsha Panchal ◽  
Apurva Patel ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Western India ◽  
Type Ii ◽  
Hematopoietic Stem ◽  
Congenital Dyserythropoietic Anemia ◽  
Cda Ii

The most frequent form of congenital dyserythropoiesis (CDA) is congenital dyserythropoietic anemia II (CDA II). CDA II is a rare genetic anemia in humans, inherited in an autosomally recessive mode, characterized by hepatosplenomegaly normocytic anemia and hemolytic jaundice. Patients are usually transfusion-independent except in severe type. We are here reporting a case of severe transfusion-dependent type II congenital dyserythropoietic anemia in a 5-year-old patient who has undergone allogeneic hematopoietic stem cell transplantation (HSCT) at our bone marrow transplantation centre. Patient has had up until now more than 14 mL/kg/month of packed cell volume (PCV), which he required every 15 to 20 days to maintain his hemoglobin of 10 gm/dL and hematocrit of 30%. His pre-HSCT serum ferritin was 1500 ng/mL and he was on iron chelating therapy. Donor was HLA identical sibling (younger brother). The preparative regimen used was busulfan, cyclophosphamide, and antithymocyte globulin (Thymoglobulin). Cyclosporine and short-term methotrexate were used for graft versus host disease (GVHD) prophylaxis. Engraftment of donor cells was quick and the posttransplant course was uneventful. The patient is presently alive and doing well and he has been transfusion-independent for the past 33 months after HSCT.

Hematopoietic stem cell transplantation in a patient with mucolipidosis type II - A five-year follow-up

2020 ◽  
Vol 129 (2) ◽  
pp. S20-S21
Author(s):  
Luise Ammer ◽  
Lohmöller Benjamin ◽  
Charlotte Aries ◽  
Mona Lindschau ◽  
Katharina Stumpfe ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Type Ii ◽  
Hematopoietic Stem ◽  
Mucolipidosis Type

Autologous hematopoietic stem cell transplantation in refractory celiac disease with aberrant T cells

Blood ◽  
2006 ◽  
Vol 109 (5) ◽  
pp. 2243-2249 ◽  
Author(s):  
Abdulbaqi Al-toma ◽  
Otto J. Visser ◽  
Hyacintha M. van Roessel ◽  
B. Mary E. von Blomberg ◽  
Wieke H. M. Verbeek ◽  
...  
Keyword(s):  
T Cells ◽  
Stem Cell ◽  
Celiac Disease ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Type Ii ◽  
Hematopoietic Stem ◽  
Refractory Celiac Disease

Abstract Autologous hematopoietic stem cell transplantation (ASCT) is an increasingly accepted treatment for refractory autoimmune diseases. Refractory celiac disease with aberrant T cells (RCD type II) is unresponsive to available therapies and carries a high risk of transition into enteropathy associated T-cell lymphoma (EATL). This study reports on the feasibility, safety, and efficacy of ASCT in patients with RCD type II. Thirteen patients with RCD type II were evaluated. Seven patients (4 men, 3 women, mean age 61.5 years [range, 51-69 years]) underwent transplantation. After conditioning with fludarabine and melphalan, ASCT was performed. Patients were monitored for response, adverse effects, and hematopoietic reconstitution. All 7 patients completed the mobilization and leukapheresis procedures successfully and subsequently underwent conditioning and transplantation. Engraftment occurred in all patients. No major nonhematologic toxicity or transplantation-related mortality was observed. There was a significant reduction in the aberrant T cells in duodenal biopsies associated with improvement in clinical well-being and normalization of hematologic and biochemical markers (mean follow-up, 15.5 months; range, 7-30 months). One patient died 8 months after transplantation from progressive neuroceliac disease. These preliminary results showed that high-dose chemotherapy followed by ASCT seems feasible and safe and might result in long-term improvement of patients with RCD type II whose condition did not respond promptly to available drugs.

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