Stem cell transplantation : home care, graft-versus-host disease and costs

Abstract

Allogeneic stem-cell transplantation (ASCT) is used to treat malignant and non-malignant diseases of the immunohematopoietic system. Results in terms of survival rate and less complications are continually improving due to better knowledge, supportive care, immunosuppression, new drugs against infections and HLA-typing techniques. In transplant centres worldwide patients undergoing ASCT are treated in protective environments such as laminar airflow rooms, plastic bubbles, or reversed isolation. However, the isolation may lead to undesired psychosocial side-effects for the patient.

The main aim of this thesis was therefore to investigate whether it was safe and feasible to treat patients undergoing ASCT during the pancytopenic phase at home instead of at the hospital. Moreover, as the ASCT and post-transplant therapies are considered to be expensive, costs of ASCT and the treatment of severe GVHD grades III-IV were evaluated.

Paper I introduces a new approach showing it to be safe to treat patients undergoing ASCT at home as much as possible during the pancytopenic phase. In paper II, home-care was offered and evaluated in a larger group of patients. The study showed that the home-care regimen resulted in significantly reduced needs of total parenteral nutrition (TPN) (p=0.01) and analgesics (p=0.05); fewer patients with acute GVHD grades II-IV were identified (p<0.01); the time to discharge was shortened (p=0.01); there was less transplant-related mortality (TRM) (p<0.01); and there was a significantly better survival (p<0.03), compared with hospital-care. Because a less acute GVHD could predispose less chronic GVHD and increase the risk for relapse, we conducted a long-term follow-up study of the patients regarding this (paper III). However, no significant differences regarding chronic GVHD and relapse rate could be found in the home-care group, compared to the hospital-care group. In paper II, home-care was showed to be less costly compared to hospital care.

In paper IV, a more thorough cost analysis was performed, were the initial and the five consecutive post ASCT yearly costs were identified. It was showed that re-transplantation (p=0.004), prophylactic use of granulocyte colony-stimulating factors post ASCT (p=0.008), acute leukemia (p=0.008) and major complications, such as GVHD. bacteremia, hemorrhagic cystitis, and veno-occlusive disease of the liver, were associated with increased costs. In contrast, reduced intensity conditioning (p=0.01) and home-care (p<0.05) reduced the costs. In paper V, survival rate and costs regarding treatment of infections and severe acute GVHD (grades III-IV) between 1975 and 2004 were analysed. Since year 1999, the survival rate in patients with GVHD grades III-IV has improved significantly (9% vs. 21%, p=0.02); however, this improvement was found to be related to high costs.

To conclude, home-care may be offered during the pancytopenic phase to patients undergoing ASCT. Home-care may reduce the risk for acute GVHD, improve survival, and reduce the costs for ASCT. Major complications are costly. This thesis forms the basis for future strategies to further improve the patient care and to limit major transplant-related complications and costs.