A study assessing the feasibility of randomization of pediatric and young adult patients between matched unrelated donor bone marrow transplantation and immune‐suppressive therapy for newly diagnosed severe aplastic anemia: A joint pilot trial of the North American Pediatric Aplastic Anemia Consortium and the Pediatric Transplantation and Cellular Therapy Consortium

2020 ◽  
Vol 67 (10) ◽  
Author(s):  
Michael A. Pulsipher ◽  
Leslie E. Lehmann ◽  
Alison A. Bertuch ◽  
Ghadir Sasa ◽  
Timothy Olson ◽  
...  
2018 ◽  
Vol 2 (15) ◽  
pp. 2020-2028 ◽  
Author(s):  
George E. Georges ◽  
Kris Doney ◽  
Rainer Storb

Abstract Treatment of severe aplastic anemia has improved significantly over the past 4 decades. This review will summarize the key areas of progress in the use of allogeneic hematopoietic cell transplantation and nontransplant immunosuppressive therapy (IST) for the treatment of aplastic anemia and then summarize the recommendations for first-line treatment. Based on recent data, we argue that guidelines for the initial treatment of patients with newly diagnosed severe aplastic anemia require revision. At the time of diagnosis, before beginning treatment, HLA typing should be done to identify a marrow donor among family members or in the unrelated donor registries, and a marrow transplant should be considered first-line therapy. The priority order of donor source for bone marrow transplantation is: (1) HLA-identical sibling, (2) HLA-matched unrelated donor, and (3) HLA-haploidentical donor if an HLA-matched unrelated donor is not rapidly available. Each of these donor marrow sources may be preferable to nontransplant IST. We make this recommendation because of the long-term persistent risk for disease relapse and secondary myelodysplastic syndrome or acute myeloid leukemia with the use of nontransplant IST for patients with aplastic anemia. In contrast, marrow transplantation is associated with high cure rates of aplastic anemia and a relatively low risk for graft-versus-host disease, with many patients now living for decades without the risk for disease recurrence or the development of clonal disorders. Implementation of this first-line treatment strategy will provide patients with severe aplastic anemia the best chance of long-term disease-free survival.


2021 ◽  
Vol 17 (1) ◽  
Author(s):  
Romy G. Younan ◽  
Roy A. Raad ◽  
Bassem Y. Sawan ◽  
Rabih Said

Abstract Background Treatment with immune checkpoint inhibitors has revolutionized cancer treatment over the past several years. Despite their clinical benefits, a wide range of immune-mediated toxicities can be observed including hematological toxicities. Although, the majority can easily be managed, immune-mediated adverse events rarely can be severe and difficult to approach. Herein, we are reporting a case of very severe aplastic anemia secondary to ipilimumab (I) and nivolumab (N) treatment that failed various treatment including intensive immune suppressive therapy. Case presentation We described a case of a 45-year old white male, heavy smoker presented to the clinic complaining of left flank pain. He was found to have a metastatic renal cell carcinoma for which he was treated with dual immunotherapy and later complicated by severe immune related adverse events. The patient later died after failing intensive immune suppressive therapy. Conclusion Immunotherapy has become an established pillar of cancer treatment improving the prognosis of many patients with variant malignancies. Yet, lethal adverse events can occur in rare cases. It is our duty, as physicians, to remain alert and cautious.


2017 ◽  
Vol 70 (suppl. 1) ◽  
pp. 63-65
Author(s):  
Marija Elez ◽  
Lavinika Atanaskovic ◽  
Svetlana Mirosavljevic ◽  
Gordana Ostojic ◽  
Biljana Todoric-Zivanovic ◽  
...  

Introduction. Allogeneic stem-cell transplantation is only potentially curative therapy for variety of hematology malignancies, such as acute and chronic leukemia, myelodisplastic syndrome and aplastic anemia, but also promising treatment option for other disorders. If we know that only 25% of patients have an human leukocyte antigen identical sibling donor, it is obvious that matched unrelated donor hematopoietic stem cell transplantation is an alternative for the rest of the patients. Material and Methods. Since 2013, matched unrelated donor hematopoietic stem cell transplantation has been performed routinely in the Military Medical Academy. Results. We hereby present the outcome after 77 procedures in 75 patients. Considering primary diseases, 35 patients had acute myeloid leukemia, 25 patients had acute lymphoid leukemia, 5 patients had chronic myeloid leukemia, 9 patients had myelodisplastic syndrome and we performed the transplant on 1 patient with chronic lymphocyte leukemia, 1 patient with aplastic anemia and 1 patient with T lymphoblastic lymphoma. Conclusion. It is difficult to make clear conclusions based on this heterogeneous group of patients, but it seems that these results are encouraging. Future research will be performed to evaluate matched unrelated donor and identical sibling hematopoietic stem cell transplantation in the homogenous groups with respect to primary diseases.


2017 ◽  
Vol 9 (2) ◽  
pp. 127-128
Author(s):  
Anuja Nanda ◽  
Vineeta Gupta ◽  
Prachi Maheshwari ◽  
Archna Tandon

ABSTRACT Aplastic anemia is a rare disease caused by destruction of pluripotent stem cells in bone marrow. The etiology may be radiation exposure, chemotherapy, environmental toxins or it could be autoimmune. During pregnancy it can be life threatening for both mother and child. Treatment options are erythrocytes and platelet transfusions and immune suppressive therapy. We report a series of obstetric events in the life of a woman whose pregnancy was complicated due to aplastic anemia but her subsequent obstetric outcome improved after successful bone marrow transplantation. How to cite this article Gupta V, Maheshwari P, Tandon A, Nanda A. Obstetric Outcome in a Patient with Aplastic Anemia. J South Asian Feder Obst Gynae 2017;9(2):121-122.


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