Safety and Feasibility of Rehabilitation Interventions in Children Undergoing Hematopoietic Stem Cell Transplant With Thrombocytopenia.

OBJECTIVE: To analyze the relation between platelet counts, intensities of physical therapy (PT) and occupational therapy (OT) services received, and frequencies of bleeding complications in children undergoing hematopoietic stem cell transplant (HSCT) during a period of severe thrombocytopenia. DESIGN: Retrospective review study. SETTING: Tertiary care hospital. PARTICIPANTS: Children (N=63; age, <18y) hospitalized for HSCT in 2010 and 2011 who received PT and OT services while markedly thrombocytopenic (platelet count, ≤50K/mcL). INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Intensities of PT and OT interventions, patients' platelet counts on specific therapy days, and any bleeding events (minor or major) that occurred during or shortly after rehabilitation interventions. RESULTS: Sixty-two patients (accounting for 63 HSCTs) met the criteria for analysis. Fifty-six of these patients (57 HSCTs) underwent PT and/or OT while markedly thrombocytopenic. There was no correlation between platelet counts and intensities of rehabilitation interventions. There were no major bleeding events. There was no association between minor bleeding events and intensities of PT or OT interventions and no association between minor bleeding events and platelet counts. Only 5 minor bleeding events occurred during or after moderate or intensive therapy out of 346 PT and OT sessions (1.5%). CONCLUSIONS: The results of our study suggest that bleeding complications during or after mobilization and supervised exercise during PT and OT in children with severe thrombocytopenia undergoing HSCT are minor and relatively rare. These are encouraging results for both patients and rehabilitation specialists treating this population who is at high risk of developing immobility-related complications.

Allogeneic hematopoietic stem cell transplantation for nonmalignant hematologic disorders using chemotherapy-only cytoreductive regimens and T-cell-depleted grafts from human leukocyte antigen-matched or -mismatched donors.

Nonmalignant hematologic disorders (NMHD) of childhood comprise a variety of disorders, including acquired severe aplastic anemia and inherited marrow failure syndromes. Patients with high-risk NMHD without matched related donors fare poorly with allogeneic hematopoietic alternative donor stem cell transplantation (allo-HSCT) and are at high risk for developing graft-versus-host disease following unmodified grafts. The authors retrospectively analyzed data on 18 patients affected by NMHD, lacking a human leukocyte antigen (HLA)-identical sibling donor, who underwent an alternative donor allo-HSCT at their institution between April 2005 and May 2013. Fifty percent of the patients had received prior immunosuppressive therapy, 72% had a history of infections, and 56% were transfusion dependent at the time of transplant. Cytoreduction included a combination of 3 of 5 agents: fludarabine, melphalan, thiotepa, busulfan, and cyclophosphamide. Grafts were T-cell depleted. All evaluable patients engrafted. Five died of transplant complications. The cumulative incidence of graft-versus-host disease was 6%. No patient had recurrence of disease. Five-year overall survival was 77%. Age at transplant <6 years was strongly associated with better survival. Based on these results, transplant with chemotherapy-only cytoreductive regimens and T-cell-depleted stem cell transplants could be recommended for patients with high-risk NMHD, especially at a younger age.