1.
Hematopoietic Stem Cell Transplantation in Congenital Dyserythropetic Anemia Type II: A Case Report and Review of the Literature.
Uygun, V, Russo, R, Karasu, G, Daloğlu, H, Iolascon, A, Yeşilipek, A
Journal of pediatric hematology/oncology. 2020;(6):e507-e510
Abstract
Currently, there is no guideline for the treatment of patients with congenital dyserythropoietic anemia (CDA) type II. One approach is to follow-up patients with transfusions, on the basis of individually determined target hemoglobin levels, and iron chelation according to the thalassemia guidelines. In some transfusion-dependent CDA II patients, splenectomy reduces the number of transfusions; however, the only known curative option for CDA II patients is hematopoietic stem cell transplantation (HSCT). Only a few published case reports of allogeneic HSCT in CDA II patients are available. Here, we review the literature and add our data of a CDA II patient who developed transfusion dependence and was cured with HSCT.
2.
Meeting the needs of CML patients in resource-poor countries.
Malhotra, H, Radich, J, Garcia-Gonzalez, P
Hematology. American Society of Hematology. Education Program. 2019;(1):433-442
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Abstract
Subsequent to the development and global availability of BCR/ABL-targeted tyrosine kinase inhibitors (TKIs), the prognosis of patients with chronic myeloid leukemia (CML), at least those in the chronic phase, has markedly improved, and in the developed world, the average lifespan of these patients is now close to that of age- and sex-matched subjects without the disease. However, the situation in low- and middle-income countries (LMICs) may not be so rosy. Many important differences in hematological cancers, including CML, have been highlighted in various publications in LMICs vs developed countries. These include differences in incidence and prevalence rates, age and stage of disease at diagnosis, response rates, and survival. Some of the possible reasons proposed for these are varying socioeconomic milieu (impacting availability of effective drugs and essential monitoring), environmental factors (mainly exposure to viral infections and pesticides), nutritional factors with interplay of malnutrition and diet on drug absorption and blood levels, and possible unknown genetic factors. Although generic first-generation TKIs (imatinib) are available in many parts of the world, several challenges remain in providing optimal treatment to patients with CML in resource-poor countries. Some of these include availability of optimal and high-quality BCR/ABL testing, availability and expense related to use of second- and third-generation TKIs (nilotinib, dasatinib, bosutinib, and ponatinib) and hematopoietic stem cell transplantation, issues with compliance and toxicities of drugs, and ensuring a minimal standard-of-care treatment and monitoring for every patient diagnosed with CML. For the purpose of this article, the more objective country label-LMIC-coined by the World Bank will be used (gross national income per capita between $1026 and $3995; World Bank, June 2019). Some of these issues will be discussed in this article in greater detail by experts in the field in 3 different but interconnected sections.
3.
Thiamine deficiency following umbilical cord blood transplant.
Trueg, A, Borho, T, Srivastava, S, Kiel, P
Nutrition in clinical practice : official publication of the American Society for Parenteral and Enteral Nutrition. 2013;(2):223-5
Abstract
A case of Wernicke encephalopathy (WE) in the setting of umbilical cord blood transplant is reported. The patient, originally diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia, proceeded to an umbilical cord blood transplant. He presented with altered mental status 149 days following transplant. Prompt magnetic resonance imaging, baseline thiamine level, and immediate intravenous replacement allowed for recovery of most symptoms. Accompanying this case is a review of the known cases of WE in hematopoietic cell transplants and their causative factors. This is the first known adult case of WE independent of parenteral nutrition use in the setting of umbilical cord transplant.