Re: Management of sickle cell disease in the community
The article identifies future research areas such as the discovery and development of drugs designed to treat sickle cell disease, including those that promote haemoglobin F synthesis, prevent red cell dehydration, and inhibit HbS polymerization.
However working towards development of new drugs keeping these markers as target may be complete wrong direction. Currently used Hydroxyurea increases fetal hemoglobin (HbF) and improve the clinical course of sickle cell disease (SCD) patients. However, several issues of myelo-suppression, low WBC count and reticulocytes, low level of patient compliance ( due to cost or side effects), including differences in patients' drug clearance with hydroxyurea therapy remain unresolved, predictability of drug response, reversibility of sickle cell disease-related organ damage by hydroxyurea, and the efficacy of elevated HbF has been highlighted from practical observant . Two patients were reportedly succumbed to complication with SCD despite showing fivefold to tenfold increase in HbF (5% to 25%, 3% to 31%).
Clinicians have observed major SCD-related complications despite HbF levels over 20% hence suggest that the progressive vascular changes associated with SCD are unlikely to be dramatically affected by increased HbF levels . Ware et alii observed hydroxyurea-induced decreases in reticulocytes and WBC count, with rising %HbF at MTD. There may be transient action of reducing crisis with hydroxyuea use, That is why it is recommended in this article as emergency measure in crisis only, and in only those who had 2-3 episodes of crisis.
HbF as marker of efficiency has been proven inefficient in field situations; hence antisickling effect should eb the target of new drug development for SCD. Currently available drugs were developed using HbF as marker, however failure is evident by ground level situation of increasing mortality due to SCD .
In view of efficient role as anti-sickling phyto-medicines as upcoming promising therapies ( like Niprisan) which have shown potent anti- sickeling efficiency and prevention from crisis in RCTs, Cochrane review provide clue that even without affecting or giving rise in HbF or inefficient Hb, these phytomedicines may be boon for prevention and management of SCD.
Reference:
i A cautionary note regarding hydroxyurea in sickle cell disease. Vichinsky EP1, Lubin BH. Blood. 1994 Feb 15;83(4):1124-8.
ii Predictors of fetal hemoglobin response in children with sickle cell anemia receiving hydroxyurea therapy. Ware RE, Eggleston B, Redding-Lallinger R, Wang WC, Smith-Whitley K,Daeschner C, Gee B, Styles LA, Helms RW, Kinney TR, Ohene-Frempong K. Blood. 2002 Jan 1;99(1):10-4.
iv Phytomedicines (medicines derived from plants) for sickle cell
disease (Review). The Cochrane Collaboration and published in The Cochrane Library
2015, Issue 4
Competing interests:
No competing interests
11 February 2017
NEETA KUMAR
Scientist
Neeru gupta
ICMR
E-62 ANSARI NAGAR, ALLL INDIA INSTITITE OF MEDICAL SCIENCES, ANSARI NAGAR
Rapid Response:
Re: Management of sickle cell disease in the community
The article identifies future research areas such as the discovery and development of drugs designed to treat sickle cell disease, including those that promote haemoglobin F synthesis, prevent red cell dehydration, and inhibit HbS polymerization.
However working towards development of new drugs keeping these markers as target may be complete wrong direction. Currently used Hydroxyurea increases fetal hemoglobin (HbF) and improve the clinical course of sickle cell disease (SCD) patients. However, several issues of myelo-suppression, low WBC count and reticulocytes, low level of patient compliance ( due to cost or side effects), including differences in patients' drug clearance with hydroxyurea therapy remain unresolved, predictability of drug response, reversibility of sickle cell disease-related organ damage by hydroxyurea, and the efficacy of elevated HbF has been highlighted from practical observant . Two patients were reportedly succumbed to complication with SCD despite showing fivefold to tenfold increase in HbF (5% to 25%, 3% to 31%).
Clinicians have observed major SCD-related complications despite HbF levels over 20% hence suggest that the progressive vascular changes associated with SCD are unlikely to be dramatically affected by increased HbF levels . Ware et alii observed hydroxyurea-induced decreases in reticulocytes and WBC count, with rising %HbF at MTD. There may be transient action of reducing crisis with hydroxyuea use, That is why it is recommended in this article as emergency measure in crisis only, and in only those who had 2-3 episodes of crisis.
HbF as marker of efficiency has been proven inefficient in field situations; hence antisickling effect should eb the target of new drug development for SCD. Currently available drugs were developed using HbF as marker, however failure is evident by ground level situation of increasing mortality due to SCD .
In view of efficient role as anti-sickling phyto-medicines as upcoming promising therapies ( like Niprisan) which have shown potent anti- sickeling efficiency and prevention from crisis in RCTs, Cochrane review provide clue that even without affecting or giving rise in HbF or inefficient Hb, these phytomedicines may be boon for prevention and management of SCD.
Reference:
i A cautionary note regarding hydroxyurea in sickle cell disease. Vichinsky EP1, Lubin BH. Blood. 1994 Feb 15;83(4):1124-8.
ii Predictors of fetal hemoglobin response in children with sickle cell anemia receiving hydroxyurea therapy. Ware RE, Eggleston B, Redding-Lallinger R, Wang WC, Smith-Whitley K,Daeschner C, Gee B, Styles LA, Helms RW, Kinney TR, Ohene-Frempong K. Blood. 2002 Jan 1;99(1):10-4.
iii Gujarat: 20% jump in deaths due to sickle-cell anaemia. http://indianexpress.com/article/cities/ahmedabad/gujarat-20-jump-in-dea...
iv Phytomedicines (medicines derived from plants) for sickle cell
disease (Review). The Cochrane Collaboration and published in The Cochrane Library
2015, Issue 4
Competing interests: No competing interests