Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological Disorders

Study Overview
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Brief Summary
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The purpose of this study is to study the efficacy of Traditional Chinese Medicine (TCM) on anaemic and cytopenic haematological disorders including myelodysplastic syndrome (MDS), aplastic anaemia (AA), myelofibrosis (MF) and thalassemia intermedia who do not have or did not respond to available treatment options.

Official Title
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Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological Disorders

Conditions
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Myelodysplastic Syndrome (MDS), Aplastic Anaemia (AA), Myelofibrosis (MF), Thalassemia Intermedia

Intervention / Treatment
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* Drug: Chinese herbal concoction twice a day for 6 months


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: A diagnosis of MDS, AA, MF or thal based on currently recognized diagnostic criteria. Explored or undergone established therapies eg haemopoietic stem cell transplant, immunosuppressive therapy, chemotherapy, growth factors, thalidomide, hypomethylating agent or androgens, and (a) are deemed not suitable, or (b) refuse or (c) have failed therapy A preceding follow up period (without or with treatment) of 2-4 months as baseline (depending on the severity of cytopenia) before being enrolled into this study Understand the trial nature of this treatment, agree to be compliant to medication, do not self medicate and have signed informed consent Agreeable to regular blood tests and follow up marrow study as listed in schedule Exclusion Criteria: Life expectancy of shorter than one year Significant organ failure including the following Renal impairment with Cr above 200umol/L Liver impairment with serum bilirubin > 2x upper limits or transaminase >3x upper limits Escalation of treatment of introduction of new agents including growth factors, thalidomide, hypomethylating agents, immunosuppressive therapy or androgens once started on treatment with TCM is not allowed. Continuation of current therapy at same or lower doses is allowed Women during pregnancy or lactation.

Ages Eligible for Study
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Minimum Age: 13 Years
Maximum Age: 85 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Treatment with Chinese herbal concoction<br>Patients must have a marrow study to confirm diagnosis of MDS, AA or MF. MDS is classified according to the WHO criteria and scored according to IPSS. The AA group is further classified into AA, SAA or VSAA . MF is defined by the Italian criteria and risk stratified by the Lilles Scoring system. Diagnosis of thal intermedia and major is based on previously done Hb electrophoresis and severity of disease is assessed by degree of anaemia.and frequency of blood transfusions TCM diagnosis: Syndrome differentiation according to TCM theory will be assessed as a baseline by experienced TCM collaborators and classified into one of the few defined syndromes as follows Yin deficiency of spleen and kidney Yang deficiency of spleen and kidney Deficiency of both Yin and Yang Stagnation of dampness and poison in the blood Excessive heat and poison | Drug: Chinese herbal concoction twice a day for 6 months<br>* Definition of each TCM syndrome is based on TCM theory. Formulation composition is based on each defined TCM syndrome with addition or removal of herbs as indicated by the patient's manifestation<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants with Adverse Events as a Measure of Safety and Tolerability | The following parameters will be monitored serially symptoms : ie reflection of any subjective symptoms that may be due to the treatment Serial biochemistry ( urea, electrolyte, creatinine, liver function test ) will be done at baseline, one week into the study and then every 7-8 weekly till end of the 6 months. This will monitor for any organ toxicity | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Quality of life | Quality of life (QOL) will be measured based on the EORTC QLQ C30. THis will be taken at enrolment and at 2 monthly intervals | 6 months | 
| Haematological improvement | The haematological response crietria is based on those published as follows For MDS : Report of an international working group to standardize response criteria for myelodysplastic syndromes. Blood 2000 Dec 1;96(12):3671-4. For MF : Response criteria for myelofibrosis with myeloid metaplasia: results of an initiative of the European Myelofibrosis Network (EUMNET). Blood 2005 Oct 15;106(8):2849-53. For AA : Aplastic Anemia, Pathophysiology and Treatment. Cambridge: Cambridge University Press; 2000. | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Chronic cytopenic haematological diseases, Chinese herbal concoction, Supportive management