paroxysmal nocturnal hemoglobinuria (PNH) | Page 18 | Aplastic Anemia and MDS International Foundation

paroxysmal nocturnal hemoglobinuria (PNH)

The clinical relevance of minor paroxysmal nocturnal hemoglobinuria clones in refractory cytopenia of childhood - a prospective study by EWOG-MDS.

Author(s): 
Aalbers AM, van der Velden VH, Yoshimi A, Fischer A, Noellke P, Zwaan CM, Baumann I, Beverloo HB, Dworzak M, Hasle H, Locatelli F, De Moerloose B, Göhring G, Schmugge M, Stary J, Zecca M, Langerak AW, van Dongen JJ, Pieters R, Niemeyer CM, van den Heuvel
Primary Author: 
Aalbers AM
Journal Title: 
Leukemia
Original Publication Date: 
Jun 2013

No abstract available.

Predictors of Hemoglobin Response to Eculizumab Therapy in Paroxysmal Nocturnal Hemoglobinuria

Author(s): 
Dezern AE, Dorr D, Brodsky RA
Primary Author: 
Dezern AE
Journal Title: 
Eur J Haematol
Original Publication Date: 
Oct 2012

Paroxysmal nocturnal hemoglobinuria is a clonal, hematopoietic stem cell disorder that manifests with hemolytic anemia and bone marrow failure. Eculizumab has been shown to improve anemia, decrease intravascular hemolysis and reduce the risk of thrombosis.
DESIGN AND METHODS:

This is a retrospective, single-center study of patients treated with Eculizumab and categorized according to response criteria. Complete response was defined as transfusion independence with normal hemoglobin for age/sex, absence of symptoms, and lactate dehydrogenase < 1.5 times the upper limit of normal. A good partial response was defined as decreased transfusions from pretreatment and lactate dehydrogenase <1.5 upper limit of normal without thrombosis. These patients did not achieve normal hemoglobins for age and sex. A suboptimal response was defined as unchanged transfusion needs and persistent of symptoms.
RESULTS:

Caregivers

Over 65 million Americans take care of other adults, often parents or spouses, or children with special medical needs. Caregivers may be a day-to-day lifeline for patients, or they may support them from afar in otherways. These generous people need support to understand how they can best play their role.