Traditional Chinese herbal medicine in the supportive management of patients with chronic cytopaenic marrow diseases -- a phase I/II clinical study.

We report on a phase I/II, single arm clinical trial studying the safety and efficacy of Traditional Chinese Medicine (TCM) in patients with various chronic cytopaenic marrow diseases including myelodysplastic syndrome (MDS), myelofibrosis (MF), aplastic anaemia (AA) and thalassemia intermedia, who either have failed, are unfit for or refused currently available Western medical treatment. Patients took oral herbal concoctions according to their TCM syndromes for 24 weeks while continuing with western medical management. The median age of this group of 31 patients was 61 (26--84) years old and median disease duration was 5 years (0.3--40 years). TCM herbs were well tolerated in these patients with multiple comorbidities and previous disease-related complications. Twenty-three patients completed the study with 5 (2 with MDS, 2 with MF and 1 with SAA) achieving some degree of haematological improvement. EORTC quality of life indicators improved in more than half of patients. This small study offers positive results and provides the basis for future larger studies which should randomize patients with MDS, MF and AA managed with standard Western medical treatment to without and with upfront combinations with TCM herbs. This will conclusively define the role of TCM in the supportive management of these diseases. This study was registered with Clinicaltrial.gov as NCT01224496.