Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality

Abstract

A prospective randomized trial of therapy for severe aplastic anemia was designed to compare early bone marrow transplantation with conventional treatments. All patients with a sibling matched at the major histocompatibility region were transplanted. Transplantation was performed with 17–100 (median 33) days of original diagnosis. Conventional treatments included transfusion support with or without androgens. Twenty-four of 36 patients intered on the transplant arm are alive after 4–20 (median 9) mo with full marrow reconstitution. Only two are limited by chronic graft-versus-host disease. In contrast only 12 of 31 conventionally treated patients are alive. Six of these survivors have improved, five incompletely. The 19 nontransplant deaths have occurred within 1–11 (median 3) mo of diagnosis. Compared to nontransplant regimens, early transplantation more effectively restores normal marrow function and decreases the acute mortality of severe marrow aplasia (p = 0.006). Pending longer follow-up, early marrow transplantation appears to be the most effective available treatment for severe aplastic anemia.

Changing prognosis in paroxysmal nocturnal haemoglobinuria disease subcategories: an analysis of the International PNH Registry

“…While significant advances have been reported in the detection of the GPI-AP-deficient cells, as well as in the pathophysiology of the disease, the natural history and the identification of survival prognostic factors in PNH have been rarely described. 8,10,13,15,19 From earlier descriptions, the clinical spectrum of PNH has resulted in two described forms of presentation: one form, predominantly haemolytic without overt marrow failure, referred to as classic PNH 1-6 (and reviewed in Young et al 4 and Parker et al 14 ), and the other one with marrow failure, often described as the AA-PNH syndrome 29 (and reviewed in Young et al 4 and Parker et al 14 ). This dichotomy was used as a working classification but did not really encompass the clinical spectrum of the disease.…”

Increased apoptotic cells in bone marrow biopsies from patients with aplastic anaemia

“…Posterior iliac crest bone marrow biopsies were obtained from 11 patients with AA aged 16-65 years (five females and six males). Four had severe and seven moderate aplastic anaemia (Camitta et al, 1976). The values (median, range) of haemoglobin, neutrophil and platelet counts were 5 .…”

Comparable survival outcome between transplantation from haploidentical donor and matched related donor or unrelated donor for severe aplastic anemia patients aged 40 years and older: A retrospective multicenter cohort study

“…All patients met Camitta's criteria for acquired SAA or vSAA. 13 The study is an extension of clinical trial No. ChiCTR-ONC-12002107, which is registered in the Chinese Clinical Trial Registry.…”