Bone Marrow Failure Syndromes (BMFS)
are a group of medical disorders in which there is a paucity of RBCs, WBCs and
Platelets in the blood either singly or all together (pancytopenia). These conditions
may be either hereditary or acquired. Syndromes that result in pancytopenia
include the following: Fanconi Anemia and Dyskeratosis Congenita. Syndromes
usually limited to single hematopoietic lineage include the following: Severe Congenital
Neutropenia (SCN), including Kostmann syndrome, Diamond-Blackfan Anemia, Shwachman-Diamond
Syndrome, Congenital Amegakaryocytic Thrombocytopenia (CAMT), and Thrombocytopenia
Absent Radii (TAR) syndrome.
BMFS are usually
caused by dysfunctional stem cell production, defective growth factors, a
dysfunctional environment, defective nutrition, accelerated cell death, and in
the case of acquired aplastic anemia, immune-mediated abnormalities. Severe
anemia can cause fatigue and cardiac failure; low leukocyte count can increase
susceptibility to infections; and low platelet count can cause spontaneous
bleeding. Several inherited BMFS are
associated with a high risk of leukemia and solid tumours.
Diamond-Blackfan
anemia is a pure red cell aplasia, usually diagnosed in the first year.
Physical abnormalities may or may not be present. Most patients improve with
corticosteroid treatment, failing which, red cell transfusions with iron
chelation, and lastly, stem cell transplant may be tried. Shwachman–Diamond
syndrome (SDS), or Shwachman–Bodian–Diamond
syndrome, is a rare
congenital disorder characterized
by exocrine pancreatic insufficiency, bone marrow dysfunction, skeletal
abnormalities and short stature.Treatment of SDS includes
pancreatic enzyme replacement, intravenous antibiotics, transfusions of RBCs,
platelets and granulocyte colony stimulating factor (G-CSF), and orthopedic
surgery as required.
TAR is diagnosed
exclusively in the neonatal period. Treatment is with platelet transfusions.
Surgery and stem cell transplantation are used as required. CAMT presents with
isolated thrombocytopenia in infancy and develops into pancytopenia in later
childhood. It has tendency to transform into Myelodysplastic syndrome (MDS) or
acute myeloid leukemia (AML). The primary treatment is bone marrow
transplantation. SCN is defined as early onset severe neutropenia, with most patients
developing MDS or AML. Patients often improve when treated with G-CSF; however,
annual bone marrow assessment is mandatory to check for serious side effects.
Fanconi
anemia is one of the most common inherited BMFSs, with nearly 75% patients
exhibiting some physical abnormalities. Standard treatment consists of need
based transfusion with RBC and platelets, antibiotics, HLA-matched donor stem
cell transplant, and treatment with androgens. Patients in the long run may
develop leukemia, MDF and solid tumours. Dyskeratosis congenita is a rare and
progressive congenital disorder with characteristic features of skin
hyperpigmentation, nail dystrophy and oral leukoplakia. Treatment is with
androgens, with the addition of erythropoietin, and G-CSF. The overall results
of bone marrow transplant are poor. About 15 % of patients are likely to
develop cancer.
Acquired
aplastic anemia is a BMFS which can be life threatening if left untreated.
Exposure to chemicals and drugs, pregnancy, immune dysfunction, and a genetic
predisposition are believed to cause this condition. It can lead to other
disorders like paroxysmal nocturnal hemoglobinuria (PNH), MDS, and AML.
Treatment options include immune suppressive therapy with antithymocyte globulin
(ATG) and cyclosporine (CsA) or high–dose cyclophosphamide. With the
availability of a suitable donor, hematopoietic stem-cell transplantation
offers an opportunity for cure.
Almost
all BMFS have a serious prognosis, with a magnified long term risk for
malignancies. Specific treatments are available which may positively impact the
course of the disease; however, most are associated with serious long term side
effects. In this scenario, Ayurvedic herbal medicines offer an alternative
treatment option which can be safely used for long periods both in children as
well as adults and helps keep the blood counts stable at acceptable levels.
The
therapeutic action of Ayurvedic herbal medicines in BMFS is at multiple levels.
Some medicines stimulate the bone marrow and help accelerate the proliferation
and differentiation of the hematopoietic system. Some medicines help normalize
specific and general tissue metabolism as well as metabolite nutrition
channels. Herbal medicines and herbomineral compounds help provide an optimum
environment which gradually helps dilute the deleterious effect of
dysfunctional genes and slows down the death rate of healthy cells. Ayurvedic
herbal medicines also help optimize a faulty immune system so that it gradually
starts working in favor of the body systems and not against them.
Depending
upon the type of BMFS and the presenting severity, different permutations and
combinations of Ayurvedic herbal medicines may be required for effective
treatment and lasting results. Modifications in the medical protocol and dosage
may also be required as per patient feedback and observed clinical effects. An average of six to twelve months of regular
treatment may be required to stabilize the blood picture in a typical adult
patient; further treatment decisions need to be taken on an individual
case-to-case basis.
Ayurvedic
treatment can thus help stabilize and maintain patients with BMFS, both in
adults as well as in children. In addition to a very low risk profile for side
effects, Ayurvedic herbal treatment can cost just a fraction of the cost of
modern treatment. Because of the serious nature and poor long term prognosis of
BMFS, with the potential for long term complications and high probability of
cancer, all patients taking Ayurvedic herbal treatment should be under the concurrent
regular supervision of several health
professionals, including a hematologist, and a general physician.
The writer, Dr A A Mundewadi, is available as an
Ayurvedic consultant at https://www.mundewadiayurvedicclinic.com and http://www.ayurvedaphysician.com
