Hemoglobino characteristic were investigated in 492 blood specimens collected in a small island community (1,450 inhabitants) in the Bahamas. Using two different methods, abnormal variants were detected in 20.3% of the sample, including genotypes AS, AC, AF(A/HPFH), SC, and SF. Biodemographic evidence suggests that the origin and distribution of the hemoglobinpathies in this population have been influenced by historical migrations and genetic drift. The opportunity for drift has been particularly heightened by the "founder effect," the maintenance of small population size, a relatively endogamous mating structure, and restricted immigration. Health survey results reveal a wide discrepancy between the actual prevalence of abnormal hemoglobins and that indicated by informant reports.
Cord blood screening of newborns for sickle cell disease was initiated in Florida at the Miami Sickle Cell Clinical Center in 1979 as a part of its participation in the national Cooperative Study of Sickle Cell Disease. Funding for newborn sickle cell screening in Florida began as a pilot project with a line-item appropriation in fiscal years 1983/84, and this funding for the three medical schools (University of Florida, University of Miami, and University of South Florida) continues to the present. During fiscal year 1984/85, a contract was negotiated between the University Hospital at Jacksonville and the local Children's Medical Services' office for the provision of newborn sickle cell screening. This contract has been renewed each year to the present. The primary objective in each of these programs is to identify babies with clinically significant hemoglobinopathies and to offer the services of the institution's pediatric hematology division. Additionally, education, genetic counseling, family studies, and appropriate referrals are provided for all babies found to have any hemoglobinopathy and for their family members. Initially, at the University of Miami, cord blood specimens were obtained at the time of delivery from at-risk (mostly black) mothers from whom informed consent had been obtained during a visit to one of several prenatal clinics associated with the University of Miami/Jackson Memorial (UM/ JM) Medical Center. It was soon discovered that many at-risk expectant mothers did not attend a prenatal clinic before their babies were born. Our internal review board did not consider consent "at delivery" informed consent.
of P e d i a t r i c s and Urology, Boston. We have p r e v i o u s l y described r e s u l t s w i t h a novel form o f adoptive immunotherapy using i n v i t r o immunized ( I V I ) autologous lymphocytes. We now r e p o r t r e s u l t s from an on-going study using an improved v e r s i o n of t h a t method. P a t i e n t lymphoc y t e s a r e d e p l e t e d o f suppressor T -c e l l s and i n v i t r o immunized f o r 60 hours i n medium c o n t a i n i n g autologous serum, mixed lymphoc y t e c u l t u r e s u p e r n a t a n t and a 3M KC1 a.atologous twnor e x t r a c t . The r e s u l t a n t I V I c e l l s a r e washed, d e p l e t e d again of suppressor c e l l s and i n f u s e d i n t o t h e p a t i e n t . A l l p a t i e n t s a l s o r e c e i v e c i m e t i d i n e t o block i n vivo suppressor c e l l s . P a t i e n t s w i l l be t r e a t e d monthly f o r 6 months. To d a t e , 30 p a t i e n t s have received a t o t a l o f 113 c e l l i n f u s i o n s , a l l d e l i v e r e d on an o u t p a t i e n t b a s i s , (average of l o 9 I V I c e l l s / i n f u s i o n ) . Toxicity has been minimal, c o n s i s t i n g o n l y of mild, t r a n s i e n t f e v e r and c h i l l s accompanying 42 of t h e i n f u s i o n s ( 3 7 % ) . Twenty-one p a t i e n t s have reached t h e f i r s t e v a l u a t i o n t i m q o i n t a t 3 months. (M~=minor response;PR=partial response;CR=complete response) This approach t o immunotherapy is s a f e and appears t o be b i o l o g i c a l l y a c t i v e , i n t h a t 7 of 21 evaluable p a t i e n t s (33%) have evidence of s t a b l e d i s e a s e o r o b j e c t i v e response a f t e r only 3 months of t r e a t n e n t . We have p r e v i o u s l y described t h a t thymic hormones might h e u s e f u l i n t h e treatment of h i s t i o c y t o s i s -X . Based on t h o s e d a t a , t h e O f f i c e o f Orphan Products Development a t t h e FDA i s sponsoring a randomized, double-blind, placebo-controlled c l i n i c a l s t u d y t o determine i f Suppressin A, a mixture of thymic hormones t h a t s p e c i f i c a l l y induce t h e d i f f e r e n t i a t i o n o f suppress o r T-lymphocytes, i s b i o l o g i c a l l y a c t i v e i n histiocytosis-X. P a t i e n t s a r e randomized t o r e c e i v e e i t h e r Suppressin A o r placebo f o r 42 days, a f t e r which they a r e re-evaluated and t h e codebroken. To h e l p i n t h e e v a l u a t i o n of b i o l o g i c a l a c t i v i t y , f o u r d i s t i n c t c l i n i c a l s c o r i n g systems were c r e a t e d : 1)organ s p e c i f i c ; 2)whole body; 3 ) v i t a l systems ( r e s p i r a t o r y , h e p a t i c and hematopoetic f u n c t i o n ) ; and 4 ) o v e r a l l performance s t a t u s . The r e s u l t s t o d a t e from t h e i n i t i a l 1 3 p a t i e n t s who have completed t h e six-week t r e a t m e n t course i n t h i s on-going study a r e : Scoring System Suppressin A (N=8) TREATMENT OF HISTIOCYTOSIS-X WITH SUPPRESSIN- t a i n d i c a t e b i o l ...
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