The life of patients with thalassemia major

Borgna‐Pignatti, Caterina

doi:10.3324/haematol.2009.017228

Cited by 110 publications

(126 citation statements)

References 20 publications

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“…Improved availability of medical services, growing knowledge of the pathogenesis of disease symptoms, novel pharmaceutics and advancing technology are all responsible for the longevity of patients with the hereditary hemoglobinopathies. In Italy 60% of the patients with TM in 2009 were above 30 years of age [1], while in Greece the probability of survival was 65% at the age of 50 years [2]. Similarly, in SCD the survival has greatly improved; in 1994 the median survival for males with sickle cell anemia was 42 years and for females 48 years [3], while one decade later the Dallas cohort reported 85.6% overall survival [4].…”

Section: Introductionmentioning

confidence: 99%

Thalassemia Major and Sickle Cell Disease in Adolescents and Young Adults

Yacobovich

Tamary

2014

Acta Haematol

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The increased longevity of patients with thalassemia and sickle cell disease (SCD) introduces new clinical challenges due to the accumulation of disease-related morbidity, psychosocial issues and health care adjustments. Patients with thalassemia major now live into adulthood without suffering heart failure but must confront delayed puberty, impaired fertility and progressive bone disease. The increased survival in SCD brings to the front previously unrecognized complications including pulmonary hypertension, silent cerebral infarcts and also reproductive dysfunction. Adolescents and young adults (AYAs) have age-related psychosocial needs in their transition from the pediatric health care environment to the adult system. In this review we present the uniquely age-related medical issues facing the AYA thalassemia and SCD cohort in their transition into adulthood.

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Section: Introductionmentioning

confidence: 99%

Thalassemia Major and Sickle Cell Disease in Adolescents and Young Adults

Yacobovich

Tamary

2014

Acta Haematol

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“…Around 300,000 children are born with sickle cell disease each year , and there are around 36. million people infected with HIV [48]. These conditions result in a reduced life expectancy and quality of life [48][49][50][51][52][53]]. …”

Section: • Metachromatic Leukodystrophymentioning

confidence: 99%

Umbilical Cord Blood Hematopoietic Stem and Progenitor Cell Expansion for Therapeutic Use

Watt¹,

Hua²

2017

Umbilical Cord Blood Banking for Clinical Application and Regenerative Medicine

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Hematopoietic stem cell transplantation (HSCT) is a potentially curative therapy for severe hematological malignancies and other severe disorders of the blood, immune system, and bone marrow. It is the most successful regenerative therapy to date, with 2013 marking the one millionth HSCT and the 25th anniversary of the irst umbilical cord blood (UCB) HSCT. UCB has most often been used for allogeneic HSCT when a matched bone marrow or peripheral blood donor is unavailable. Recently, novel genome editing technologies to correct inherited gene disorders or to modulate biomarkers/ receptors on HSC and the potential use of HSCT for a variety of other nonmalignant conditions have led to a surge of interest in autologous HSCT, with the HSC source depending on the condition to be treated. UCB HSCs may be used to generate red blood cells, granulocytes, or platelets ex vivo for transfusion into diicult-to-transfuse patients. Alternatively, UCB may be reprogrammed to induced pluripotent stem cells or used to generate cell lines, which can then be diferentiated into diferent cell lineages for transfusion or used as diagnostic reagents. Disadvantages of UCB are its restricted cell numbers and delayed hematological engraftment. Here, UCB HSC expansion/ manipulation ex vivo and clinical applications are addressed.

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“…Merely 50 to 65% of patients live beyond the age of 35 years even after having transfusions, that too in developed or high-income countries. [47][48][49] In order to overcome these complications, medications for removing excessive iron overload become an important requirement.…”

Section: Blood Transfusionsmentioning

confidence: 99%