title: Health and functional status of long-term survivors of bone marrow transplantation. EBMT Working Party on Late Effects and EULEP Study Group on Late Effects. European Group for Blood and Marrow Transplantation.
abstract: BACKGROUND
  Although many patients now survive the short-term complications of bone marrow transplantation for life-threatening hematologic disease, information on the health and activity of long-term survivors is sparse.
  
  
  OBJECTIVE
  To evaluate the morbidity and mortality of patients surviving more than 5 years after allogeneic bone marrow transplantation.
  
  
  METHODS
  Retrospective, multicenter study.
  
  
  METHODS
  798 recipients of bone marrow transplants (477 adults, 321 children) from 43 European centers. Patients had received transplants before December 1985 and had survived at least 5 years. Patients had received allogeneic or syngeneic bone marrow for leukemia, lymphoma, inborn diseases of the hematopoietic and immune systems, and severe aplastic anemia.
  
  
  METHODS
  Survival, clinical performance according to Karnofsky score (in increments of 10%), and social reintegration were assessed as outcomes. Patient age and sex, primary disease and status at transplantation, histocompatibility of the donor, conditioning regimen, type of prophylaxis of graft-versus-host disease, and acute and chronic graft-versus-host disease were evaluated as variables.
  
  
  RESULTS
  For the 55 5-year survivors, actuarial mortality was 8% at 10 years and 14% at 15 years. The leading causes of death were disease recurrence (21 patients), chronic graft-versus-host disease with complicating infections and lung disease (11 patients), secondary cancer (8 patients), and the acquired immunodeficiency syndrome (AIDS) (5 patients). When patients with recurrent disease were excluded, late death was associated with chronic graft-versus-host disease (P < 0.001), occurrence of secondary cancer (P < 0.001), male sex of the patient (P = 0.05), and female sex of the donor (P = 0.002). Clinical performance was normal (Karnofsky score, 100%) or minimally reduced (Karnofsky score, 90%) in 93% of patients; 89% of patients resumed full-time work or school. Reduced performance status and incomplete resumption of social activity were associated with chronic graft-versus-host disease, recurrent leukemia, AIDS, secondary cancer, organ dysfunction, and neurologic or psychological problems. Other risk factors for incomplete resumption of social activity were female sex (P = 0.002) and older age at transplantation (P = 0.001).
  
  
  CONCLUSIONS
  More than 5 years after bone marrow transplantation, most patients were in good health (93%) and had returned to full-time work or school (89%). Recurrence of the primary disease, secondary cancer, and chronic graft-versus-host disease and its sequelae remain problems for some patients.
authors.affiliation: University of Munich, Germany.
authors.forename: T
authors.initials: T
authors.lastname: Duell
authors.forename: M T
authors.initials: MT
authors.lastname: van Lint
authors.forename: P
authors.initials: P
authors.lastname: Ljungman
authors.forename: A
authors.initials: A
authors.lastname: Tichelli
authors.forename: G
authors.initials: G
authors.lastname: Socié
authors.forename: J F
authors.initials: JF
authors.lastname: Apperley
authors.forename: M
authors.initials: M
authors.lastname: Weiss
authors.forename: A
authors.initials: A
authors.lastname: Cohen
authors.forename: E
authors.initials: E
authors.lastname: Nekolla
authors.forename: H J
authors.initials: HJ
authors.lastname: Kolb
mesh_terms: D000328:Adult; D016026:Bone Marrow Transplantation / Q000401:mortality / Q000523:psychology; D002648:Child; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology / Q000517:prevention & control; D006304:Health Status; D006801:Humans; D007398:Interpersonal Relations; D008297:Male; D011183:Postoperative Complications; D012008:Recurrence; D012189:Retrospective Studies; D017741:Survivors; D019172:Transplantation Conditioning